Category Archives: Stem Cell Treatments

Researchers are harnessing the tools of genetic engineering to develop potential treatments for human hair loss. dNovo, a biotech startup, claims to have reprogrammed human stem cells into follicle-forming cells and transferred them into the mouse above which you can see has grown a nice tuft, albeit in an odd location. From Technology Review:

In addition to dNovo, a company called Stemson (its name is a portmanteau of "stem cell" and "Samson") has raised $22.5 million from funders including from the drug company AbbVie. Cofounder and CEO Geoff Hamilton says his company is transplanting reprogrammed cells onto the skin of mice and pigs to test the technology[]

So is stem-cell technology going to cure baldness or become the next false hope? Hamilton, who was invited to give the keynote at this year'sGlobal Hair Loss Summit, says he tried to emphasize that the company still has plenty of research ahead of it. "We have seen so many [people] come in and say they have a solution. That has happened a lot in hair, and so I have to address that," he says. "We're trying to project to the world that we are real scientists and that it's risky to the point I can't guarantee it's going to work."

image: dNovo

See the original post here:
This weird mouse with a tuft of human hair could be the future of a stem cell treatment for baldness - Boing Boing

Four years ago, a small Philadelphia biotech company won U.S. approval for the first gene therapy to treat an inherited disease, a landmark after decades of research aimed at finding ways to correct errors in DNA.

Since then, most of the world's largest pharmaceutical companies have invested in gene therapy, as well as cell therapies that rely on genetic modification. Dozens of new biotech companies have launched, while scientists have taken forward breakthroughs in gene editing science to open up new treatment possibilities.

But the confidence brought on by such advances has also been tempered by safety setbacks and clinical trial results that fell short of expectations. In 2022, the outlook for the field remains bright, but companies face critical questions that could shape whether, and how soon, new genetic medicines reach patients. Here are five:

Food and Drug Administration approval of Spark Therapeutics' blindness treatment Luxturna a first in the U.S. came in 2017. A year and a half later, Novartis' spinal muscular atrophy therapy Zolgensma won a landmark OK.

But none have reached market since, with treatments from BioMarin Pharmaceutical and Bluebird bio unexpectedly derailed or delayed.

That could change in 2022. Two of Bluebird's treatments, for the blood disease beta thalassemia and a rare brain disorder, are now under review by the FDA, with target decision dates in May and June. BioMarin, after obtaining more data for its hemophilia A gene therapy, plans to soon approach the FDA about resubmitting an application for approval.

Others, such as CSL Behring and PTC Therapeutics, are also currently planning to file their experimental gene therapies with the FDA in 2022.

Approvals, should they come, could provide important validation for their makers and expand the number of patients for whom genetic medicines are an option. In biotech, though, approvals aren't the end of the road, but rather the mark of a sometimes challenging transition from research to commercial operations. With price tags expected to be high, and still outstanding questions around safety and long-term benefit, new gene therapies may prove difficult to sell.

A record $20 billion flowed into gene and cell therapy developers in 2020, significantly eclipsing the previous high-water mark set in 2018.

Last year, the bar was set higher still, with a total of $23 billion invested in the sector, according to figures compiled by the Alliance for Regenerative Medicine. About half of that funding went toward gene therapy developers specifically, with a similar share going to cell-based immunotherapy makers.

Driving the jump was a sharp increase in the amount of venture funding, which rose 73% to total nearly $10 billion, per ARM. Initial public offerings also helped, with sixteen new startups raising at least $50 million on U.S. markets.

Entering 2022, the question facing the field is whether those record numbers will continue. Biotech as a whole slumped into the end of last year, with shares of many companies falling amid a broader investment pullback. Gene therapy developers, a number of which had notable safety concerns crop up over 2021, were hit particularly hard.

Moreover, many startups that jumped to public markets hadn't yet begun clinical trials roughly half of the 29 gene and cell therapy companies that IPO'd over the past two years were preclinical, according to data compiled by BioPharma Dive. That can set high expectations companies will be hard pressed to meet.

Generation Bio, for example, raised $200 million in June 2020 with a pipeline of preclinical gene therapies for rare diseases of the liver and eye. Unexpected findings in animal studies, however, sank company shares by nearly 60% last December.

Still, the pace of progress in gene and cell therapy is fast. The potential is vast, too, which could continue to support high levels of investment.

"I think fundamentally, investment in this sector is driven by scientific advances, and clinical events and milestones," said Janet Lambert, ARM's CEO, in an interview. "And I think we see those in 2022."

The potential of replacing or editing faulty genes has been clear for decades. How to do so safely has been much less certain, and concerns on that front have set back the field several times.

"Safety, safety and safety are the first three top-of-mind risks," said Luca Issi, an analyst at RBC Capital Markets, in an interview.

Researchers have spent years making the technology that underpins gene therapy safer and now have a much better understanding of the tools at their disposal. But as dozens of companies push into clinical trials, a number of them have run into safety problems that raise crucial questions for investigators.

In trials run by Audentes Therapeutics and by Pfizer (in separate diseases), study volunteers have tragically died for reasons that aren't fully understood. UniQure, Bluebird bio and, most recently, Allogene Therapeutics have reported cases of cancer or worrisome genetic abnormalities that triggered study halts and investigations.

While the treatments being tested were later cleared in the three latter cases, the FDA was sufficiently alarmed to convene a panel of outside experts to review potential safety risks last fall. (Bluebird recently disclosed a new hold in a study of its sickle cell gene therapy due to a patient developing chronic anemia.)

The meeting was welcomed by some in the industry, who hope to work with the FDA to better detail known risks and how to avoid them in testing.

"[There's] nothing better than getting people together and talking about your struggles, and having FDA participate in that," said Ken Mills, CEO of gene therapy developer Regenxbio, in an interview. "The biggest benefit probably is for the new and emerging teams and people and companies that are coming into this space."

Safety scares and setbacks are likely to happen again, as more companies launch additional clinical trials. The FDA, as the recent meeting and clinical holds have shown, appears to be carefully weighing the potential risks to patients.

But, notably, there hasn't been a pullback from pursuing further research, as has happened in the past. Different technologies and diseases present different risks, which regulators, companies and the patient community are recognizing.

"We're by definition pushing the scientific envelope, and patients that we seek to treat often have few or no other treatment options," said ARM's Lambert.

Last June, Intellia Therapeutics disclosed early results from a study that offered the first clinical evidence CRISPR gene editing could be done safely and effectively inside the body.

The data were a major milestone for a technology that's dramatically expanded the possibility for editing DNA to treat disease. But the first glimpse left many important questions unanswered, not least of which are how long the reported effects might last and whether they'll drive the kind of dramatic clinical benefit gene editing promises.

Intellia is set to give an update on the study this quarter, which will start to give a better sense of how patients are faring. Later in the year the company is expecting to have preliminary data from an early study of another "in vivo" gene editing treatment.

In vivo gene editing is seen as a simpler approach that could work in more diseases than treatments that rely on stem cells extracted from each patient. But it's also potentially riskier, with the editing of DNA taking place inside the body rather than in a laboratory.

Areas like the eye, which is protected from some of the body's immune responses, have been a common first in vivo target by companies like Editas Medicine. But Intellia and others are targeting other tissues like the liver, muscle and lungs.

Later this year, Verve Therapeutics, a company that uses a more precise form of gene editing called base editing, plans to treat the first patient with an in vivo treatment for heart disease (which targets a gene expressed in the liver.)

"The future of gene editing is in vivo," said RBC's Issi. His view seems to be shared by Pfizer, which on Monday announced a $300 million research deal with Beam Therapeutics to pursue in vivo gene editing targets in the liver, muscle and central nervous system.

With more and more cell and gene therapy companies launching, the pipeline of would-be therapies has grown rapidly, as has the number of clinical trials being launched.

Yet, many companies are exploring similar approaches for the same diseases, resulting in drug pipelines that mirror each other. A September 2021 report from investment bank Piper Sandler found 21 gene therapy programs aimed at hemophilia A, 19 targeting Duchenne muscular dystrophy and 18 going after sickle cell disease.

In gene editing, Intellia, Editas, Beam and CRISPR Therapeutics are all developing treatments for sickle cell disease, with CRISPR the furthest along.

As programs advance and begin to deliver more clinical data, companies may be forced into making hard choices.

"[W]e think investors will place greater scrutiny as programs enter the clinic and certain rare diseases are disproportionately pursued," analysts at Stifel wrote in a recent note to investors, citing Fabry disease and hemophilia in particular.

This January, for example, Cambridge, Massachusetts-based Avrobio stopped work on a treatment for Fabry that was, until that point, the company's lead candidate. The decision was triggered by unexpected findings that looked different than earlier study results, but Avrobio also cited "multiple challenging regulatory and market dynamics."

Read more here:
5 questions facing gene therapy in 2022 - BioPharma Dive

What are stem cells?

All of the blood cells in your body - white blood cells, red blood cells, and platelets - start out as young (immature) cells called hematopoietic stem cells. Hematopoietic means blood-forming.These are very young cells that are not fully developed. Even though they start out the same, these stem cells can mature into any type of blood cell, depending on what the body needs when each stem cell is developing.

Stem cells mostly live in the bone marrow (the spongy center of certain bones). This is where they divide to make new blood cells. Once blood cells mature, they leave the bone marrow and enter the bloodstream. A small number of the immature stem cells also get into the bloodstream. These are called peripheral blood stem cells.

Stem cells make red blood cells, white blood cells, and platelets. We need all of these types of blood cells to keep us alive. For these blood cells to do their jobs, you need to have enough of each of them in your blood.

Red blood cells carry oxygen away from the lungs to all of the cells in the body. They bring carbon dioxide from the cells back to the lungs to be exhaled. A blood test called a hematocrit shows how much of your blood is made up of RBCs. The normal range is about 35% to 50% for adults. People whose hematocrit is below this level have anemia. This can make them look pale and feel weak, tired, and short of breath.

White blood cells help fight infections caused by bacteria, viruses, and fungi. There are different types of WBCs.

Neutrophilsare the most important type in fighting infections. They are the first cells to respond to an injury or when germs enter the body. When they are low, you have a higher risk of infection. The absolute neutrophil count (ANC) is a measure of the number of neutrophils in your blood. When your ANC drops below a certain level, you have neutropenia. The lower the ANC, the greater the risk for infection.

Lymphocytesare another type of white blood cell. There are different kinds of lymphocytes, such as T lymphocytes (T cells), B lymphocytes (B cells), and natural killer (NK) cells. Some lymphocytes make antibodies to help fight infections. The body depends on lymphocytes to recognize its own cells and reject cells that dont belong in the body, such as invading germs or cells that are transplanted from someone else.

Platelets are pieces of cells that seal damaged blood vessels and help blood to clot, both of which are important in stopping bleeding. A normal platelet count is usually between 150,000/cubic mm and 450,000/cubic mm, depending on the lab that does the test. A person whose platelet count drops below normal is said to have thrombocytopenia, and may bruise more easily, bleed longer, and have nosebleeds or bleeding gums. Spontaneous bleeding (bleeding with no known injury) can happen if a persons platelet count drops lower than 20,000/mm3. This can be dangerous if bleeding occurs in the brain, or if blood begins to leak into the intestines or stomach.

You can get more information on blood counts and what the numbers mean in Understanding Your Lab Test Results.

Depending on the type of transplant thats being done, there are 3 possible sources of stem cells to use for transplants:

Bone marrow is the spongy liquid tissue in the center of some bones. It has a rich supply of stem cells, and its main job is to make blood cells that circulate in your body. The bones of the pelvis (hip) have the most marrow and contain large numbers of stem cells. For this reason, cells from the pelvic bone are used most often for a bone marrow transplant. Enough marrow must be removed to collect a large number of healthy stem cells.

The bone marrow is harvested (removed) while the donor is under general anesthesia (drugs are used to put the patient into a deep sleep so they dont feel pain). A large needle is put through the skin on the lower back and into the back of the hip bone. The thick liquid marrow is pulled out through the needle. This is repeated until enough marrow has been taken out. (For more on this, see Whats It Like to Donate Stem Cells?)

The harvested marrow is filtered, stored in a special solution in bags, and then frozen. When the marrow is to be used, its thawed and then put into the patients blood through a vein, just like a blood transfusion. The stem cells travel to the bone marrow, where they engraft or take and start to make blood cells. Signs of the new blood cells usually can be measured in the patients blood tests in a few weeks.

Normally, not many stem cells are found in the blood. But giving stem cell donors shots of hormone-like substances called growth factors a few days before the harvest makes their stem cells grow faster and move from the bone marrow into the blood.

For a peripheral blood stem cell transplant, the stem cells are taken from blood. A special thin flexible tube (called a catheter) is put into a large vein in the donor and attached to tubing that carries the blood to a special machine. The machine separates the stem cells from the rest of the blood, which is returned to the donor during the same procedure. This takes several hours, and may need to be repeated for a few days to get enough stem cells. The stem cells are filtered, stored in bags, and frozen until the patient is ready for them. (For more on this, see Whats It Like to Donate Stem Cells?)

When theyre given to the patient, the stem cells are put into a vein, much like a blood transfusion. The stem cells travel to the bone marrow, engraft, and then start making new, normal blood cells. The new cells are usually found in the patients blood in about 4 weeks.

The blood of newborn babies normally has large numbers of stem cells. After birth, the blood thats left behind in the placenta and umbilical cord (known as cord blood) can be taken and stored for later use in a stem cell transplant. Cord blood can be frozen until needed. A cord blood transplant uses blood that normally is thrown out after a baby is born. After the baby is born, specially trained members of the health care team make sure the cord blood is carefully collected. The baby is not harmed in any way. More information on donating cord blood can be found in Whats It Like to Donate Stem Cells?

Even though the blood of newborns has large numbers of stem cells, cord blood is only a small part of that number. So, a possible drawback of cord blood is the smaller number of stem cells in it. But this is partly balanced by the fact that each cord blood stem cell can form more blood cells than a stem cell from adult bone marrow. Still, cord blood transplants can take longer to take hold and start working. Cord blood is given into the patients blood just like a blood transfusion.

Some cancers start in the bone marrow and others can spread to it. Cancer attacks the bone marrow, causing it to make too many of some cells that crowd out others, or causing it to make cells that arent healthy and don't work like they should. For these cancers to stop growing, they need bone marrow cells to work properly and start making new, healthy cells.

Most of the cancers that affect bone marrow function are leukemias, multiple myeloma, and lymphomas. All of these cancers start in blood cells. Other cancers can spread to the bone marrow, which can affect how blood cells function, too.

For certain types of leukemia, lymphoma, and multiple myeloma, a stem cell transplant can be an important part of treatment. The goal of the transplant is to wipe out the cancer cells and the damaged or non-healthy cells that aren't working right, and give the patient new, healthy stem cells to start over."

Stem cell transplants are used to replace bone marrow cells that havebeen destroyed by cancer or destroyed by the chemo and/or radiation used to treat the cancer.

There are different kinds of stem cell transplants. They all use very high doses of chemo (sometimes along with radiation) to kill cancer cells. But the high doses can also kill all the stem cells a person has and can cause the bone marrow to completely stop making blood cells for a period of time. In other words, all of a person's original stem cells are destroyed on purpose. But since our bodies need blood cellsto function, this is where stem cell transplants come in. The transplanted stem cells help to "rescue" the bone marrow by replacingthe bodys stem cells that have been destroyedby treatment. So, transplanting the healthy cellslets doctors use much higher doses of chemo to try to kill all of the cancer cells, and the transplanted stem cells can grow into healthy, mature blood cells that work normally and reproduce cells that are free of cancer.

There's another way astem cell transplant can work, if it's a transplant that uses stem cells from another person (not the cancer patient). In these cases, the transplant can help treat certain types of cancer in a way other than just replacing stem cells. Donated cells can often find and kill cancer cells better than the immune cells of the person who had the cancer ever could. This is called the graft-versus-cancer or graft-versus-leukemia effect. The "graft" is the donated cells. The effect means that certain kinds of transplants actually help kill off the cancer cells, along with rescuing bone marrow and allowing normal blood cells to develop from the stem cells.

Although a stem cell transplant can help some patients, even giving some people a chance for a cure, the decision to have a transplant isnt easy. Like everything in your medical care, you need to be the one who makes the final choice about whether or not youll have a stem cell transplant. Transplant has been used to cure thousands of people with otherwise deadly cancers. Still, there arepossible risks and complications that can threaten life, too. People have died from complications of stem cell transplant. The expected risks and benefits must be weighed carefully before transplant.

Your cancer care team will compare the risks linked with the cancer itself to the risks of the transplant. They may also talk to you about other treatment options or clinical trials. The stage of the cancer, patients age, time from diagnosis to transplant, donor type, and the patients overall health are all part of weighing the pros and cons before making this decision.

Here are some questions you might want to ask. For some of these, you may need to talk to the transplant team or the people who work with insurance and payments for the doctors office and/or the hospital:

Be sure to express all your concerns and get answers you understand. Make sure the team knows whats important to you, too. Transplant is a complicated process. Find out as much as you can and plan ahead before you start.

Its important to know the success rate of the planned transplant based on your diagnosis and stage in treatment, along with any other conditions that might affect you and your transplant. In general, transplants tend to work better if theyre done in early stages of disease or when youre in remission, when your overall health is good. Ask about these factors and how they affect the expected outcomes of your transplant or other treatment.

Many people get a second opinion before they decide to have a stem cell transplant. You may want to talk to your doctor about this, too. Also, call your health insurance company to ask if they will pay for a second opinion before you go. You might also want to talk with them about your possible transplant, and ask which transplant centers are covered by your insurance.

Stem cell transplants cost a lot, and some types cost more than others. For example, getting a donor's cells costs more than collecting your own cells. And, different drug and radiation treatments used to destroy bone marrow can have high costs. Some transplants require more time in the hospital than others, and this can affect cost. Even though there are differences, stem cell transplants can cost hundreds of thousands of dollars.

A transplant (or certain types of transplants) is still considered experimental for some types of cancer, especially some solid tumor cancers, so insurers might not cover the cost.

No matter what illness you have, its important to find out what your insurer will cover before deciding on a transplant, including donor match testing, cell collection, drug treatments, hospital stay, and follow-up care. Go over your transplant plan with them to find out whats covered. Ask if the doctors and transplant team you plan to use are in their network, and how reimbursement will work. Some larger insurance companies have transplant case managers. If not, you might ask to speak with a patient advocate. You can also talk with financial or insurance specialists at your doctors office, transplant center, and hospital about what expenses you are likely to have. This will help you get an idea of what you might have to pay in co-pays and/or co-insurance.

The National Foundation for Transplants (NFT) provides fund raising guidance to help patients, their families, and friends raise money for all types of stem cell transplants in the US. They can be reached online at http://www.transplants.org, or call 1-800-489-3863.

Originally posted here:
How Stem Cell and Bone Marrow Transplants Are Used to ...

At the University of Chicago Medicine, our transplant team works side-by-side with the patient, family and referring physician before, during and after transplantation to ensure the best possible outcome. The transplant process differs from patient to patient, but generally includes:

Most patients undergoing stem cell transplantation are cared for in our dedicated unit for approximately one week before and two to three weeks after the procedure. Select patients may receive outpatient stem cell transplant care in specially designed treatment rooms within the unit. The same physicians and nurses who provide inpatient care provide outpatient care.

The stem cell transplant unit is located on the top floor of the Center for Care and Discovery and features state-of-the-art technology and thoughtful amenities:

Our stem cell transplant physicians are members of the nationally renowned UChicago Medicine Comprehensive Cancer Center,one of only two National Cancer Institute (NCI)-designated Comprehensive Cancer Centers in Chicago. It is through the Cancer Center that we participate in clinical trials of emerging therapies. In addition, we are active participants in the Alliance for Clinical Trials in Oncology and the Blood and Marrow Transplant Clinical Trials Network. Involvement in these vital research organizations gives our patients access to the most novel and exciting treatments available.

Our stem cell transplant program laboratory is specially equipped to handle all of the blood and stem cell preparation necessary for transplant, including apheresis (separation and collection of stem cells from the blood) and cryopreservation (freezing of stem cells for future use).

Leading-edge technologies in the laboratory enable us to perform complex procedures that help improve transplant outcomes. These procedures include purging of cancerous cells and purifying donor stem cells to minimize graft-versus-host disease (a serious side effect related to the use of donor cells for transplant).

Originally posted here:
The Stem Cell Transplant Process - UChicago Medicine

Next Chapters: How Northern California blood recipients are doing years after donors helped save their lives

Updated: 10:07 AM PST Jan 3, 2022

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NOW LESS THAN A WEEK AWAY. ITS YOUR CHANCE TO HELP PEOPLE WHO NEED LIFE SAVING DONATIONS AT THE HOSPIL.TA GULSTAN: FOR A SACRAMENTO FAMILY, GIVING BLOOD IS AN IMPORTANT TRADITION. AND THEY WANT TO INSPIRE OTHSER TO DO THE SAME. KCRA THREES BRANDI CUMMINGS SHOWS US, THEY HAVE DONATED THOUSANDS OF TIM.ES BRANDI: YOU ARE LOOKING AT THE NERVE CENTER OF VITALANTS PROCESSING LAB, A PLACE WHERE BLOOD PRODUCTS AREES TTED, PREPARED AND STORED READY TO BE SENT OUT TO HOSPITALS THROUGHOUT NORTHERN CALIFORA.NI >> CAN I HAVE EVERYONES ATTENTION? BRANDI: THIS THE HEARTBEAT. VITALANTS BLOOD DONATION CENTER, THIS ONE IS IN MIDTOWN SACRAMENTO. HERE DONORS ROLL UP THREI SLEEVES AND GIVE THE BLOOD COMPONENTS PEOPLE NEED TO SURVE.IV PLATELETS TO BATTLE LEUKEMIA, RED CELLS TO RECOVERRO FM SURGERY, OR PLASMA TO CONTROL BLEENGDI TH IISS THE HEART, DONORS LIKE GARY MYERS. >> I DID MY FIRST BLOOD DONATION IN COLLEGE. THAT WOULD HAVE BEEN 15.98 BRANDI: TODAY, GARY IS A LEGEND AT VITALANT. HES DONATED 2,800 TIMES. >> PEOPLE ASKED ME WHY I DONATE AND I GO BECAUSE I CAN. AND ITS REALLY NOT A LOT MORE THAN THAT. THE BLOOD SUPPLY IS NEED.ED AND ITS SOMETHING I CADON BRANDI: A HUMBLE ANSWER, BUT THE TRUTH IS GARYS DONATED REA CORD 350 GALLONS OF BLOOD PRODUCTS SAVING PERHAPS, THOUSANDS OF LIVES. >> MY FAMILY THINKS ITVES RY COOL FOR ME, ITS MORE ABOUT HOW DO I MOTIVATE OTHER PEOE?PL BRANDI: ONE OF THE PEOPLE HES MOTIVATED, HIS SON LUCAS. >> ON HIS THE DAY OF HIS 16TH BIRTHDAY DID HIS FIRST PLASMA DONATION. HE WAS A SENIOR IN HIGH SCHOOL. THE DAY BEFORE HE LEFTOR F COLLEGE HE FINISHED HIS TH10 GALLON AND WAS THE YOUNGEST 10 GALLON DONOR IN THE HIOROFST BLOODSOURCE, WHICH HAS NOW VITALANT. BRANDI: AFTER DECADEOFS DONATIONS, GARY IS SAYING GOODBYE TO SACRAMENTO VITALANT, RELOTICANG TO NEBRASKA. >> THESE WERE ALL MY FRIENDS. AND, AND MANY OF THEM I WAS HERE WHEN THEY WERE TRAING.IN ITS JUST COOL. THEY BECOME THEY BECOME FAMI.LY BRANDI: WHILE HIS TIME AT VITALANT COMES TO END GARY PLEDGES TO KEEP DONATING, AS LONG AS HE CAN, KNOWING EACH DONATIONAS H THE POTENTIAL TO SAVE A LIFE. >> HOW CAN I LOOK THE MOTHERN I THE EYE? AND KNOW I COULD HAVE DONATED AND I JUST DIDN' ANBRDI: BRANDI CUMMINGS, KCRA 3 WSNE GULSTAN: THATS AMAZING. IF YOU WANT TO MAKE A DIFFERENCE LIKE GARY AND DONATE IN THIS YEARS BLOOD DRIVE FOR LIFE, WERE URGING PEOPLE TO MAKEN A APPOINTMENT AHEAD OF TIME. THATS SO WE CAN STICK TO COVID PROTOCOLS AND PRACTICE SOCIAL DISTANCI.NG ANDREA: TO MAKE AN APPOINTMENT, HOLD YOUR CELL PHONE CAMERA UP TO THIS Q.R. CE.OD IT WILL TAKE YOU TO A LINK WITH ALL OF THE INFORMATI.ON THE BLOOD DRIVE HAPPENS JANUARY 5 THROUGH THE 8TH, AT TWO LOCATIONS IN SACRAMENTO AND ROCK

Next Chapters: How Northern California blood recipients are doing years after donors helped save their lives

Updated: 10:07 AM PST Jan 3, 2022

For more than two decades, KCRA 3 has teamed up with Vitalant to hold the Blood Drive for Life, which this year is taking place on Jan. 5-8 across two locations in Rocklin and Sacramento.

Blood donations are used to help cancer patients, newborns, sickle cell patients and organ transplant recipients. Donated blood is also used for open-heart surgeries and other situations.

While most people are able to donate blood, only 3% do.

Over the years, KCRA 3 helped tell the stories of those in need of lifesaving blood transfusions.

For this years blood drive, Vitalant checked in with some of the people weve featured in past years for an update on the next chapter of their lives.

If you would like to join other community members for this years blood drive, click here to sign up.

Tony was diagnosed at just 4 months old with the rare blood disorder hemophagocytic lymphohistiocytosis, or HLH, and a bone marrow transplant was the only cure.

It took a year to find his marrow match. His transplant happened in December 2007 in San Francisco.

Tony is now a high school student at age 16 and his mother Kasey said in an update that without generous blood donors her son wouldnt be here and thriving today.

My son received over a hundred units of blood from age 7 months to 2 1/2 years old until he received his bone marrow transplant (BMT), she said. In such a short amount of time, he needed so much blood to keep him going and to become strong enough to receive his BMT. Watching your son go through so much and feeling helpless, relying on strangers to keep your baby alive, is such a hard thing to go through. Because of their gifts, he is playing varsity volleyball in high school, looking forward to driving, going on trips with his family and hanging out with friends. He is a wonderful young man. So, I thank blood donors daily for their selfless gifts to families like ours and for the people who make that happen.

Tony's family first shared their story with KCRA 3 for the blood drive in 2010 and then in 2012 when the Bierwirths met Tony's marrow donor.

Debbie was diagnosed with Acute Myelogenous Leukemia (AML) after her 50th birthday in 2006 and needed a bone marrow transplant.

As an African American, Debbie was told it would be hard to find a match because its harder for Black patients to find a match.

She did not find a match and decided to enroll in a clinical trial at Fred Hutchinson Cancer Research Center in Seattle for an umbilical cord blood transplant where patients receive cord blood rich with blood stem cells that could potentially cure her leukemia. Patients do not need as close of a match as they do with adult blood stem cells.

The treatment worked and has become a standard treatment for thousands of people who dont have a matched donor.

After her treatment, Debbie returned to work and was able to attend President Barack Obamas inauguration in Washington, D.C.

Shes now approaching 15 years cancer-free, is retired and is grateful for life.

Debbie encourages people to donate blood, platelets, plasma and to join Be The Match marrow registry, especially those with diverse heritage and ethnicities.

"Navigating this journey in my 14th year post-stem cell transplant, life is grand and I continue to receive an abundance of blessings," she said. "My prayer is that others diagnosed with cancer receive blessings!"

Debbie first shared her story with KCRA 3 for the blood drive in 2008.

Steven was a high school senior when he was diagnosed with leukemia at age 17.

Doctors recommended a bone marrow transplant but without a family match, he faced long odds due to his Hispanic and Chinese heritage.

Steven was able to receive a marrow transplant in September 2009 but close to two years later, his leukemia returned and he needed another transplant.

He got that second marrow transplant in 2012. Steven required hundreds of blood components during both marrow transplants and has been nicknamed Ironman for all hes undergone.

Despite two hip replacements for joints that deteriorated due to cancer treatments and medications for graft vs. host disease, Steven still finds time to give back as a counselor at Camp Okizu, a volunteer with Leukemia and Lymphoma Society, and an ambassador for young adult cancer survivors with Stupid Cancer.

In January, Steven's looking forward to celebrating 10 years since his last transplant.

Id like to thank blood donors for all they do to help patients like me, he said. Ive gone through leukemia twice and two bone marrow transplants; blood donors and marrow donors helped me survive. Im forever grateful and looking forward to a healthier future.

Steven first shared his story for the blood drive in 2011 and again in 2013 because he needed a second bone marrow transplant.

Hiking in the Sierra above Echo Lake on his way to Flagpole Peak in 2004, Paul Frydendal was aware of his surroundings but confident in his ability to navigate the snowy terrain.

But he suffered an accident by falling into a snow chute, tumbling down 800 feet. By the time he stopped, he had nine broken ribs, a punctured lung, a shattered femur, a broken right arm and tailbone, a broken bone in his neck, severe head lacerations and internal injuries.

He was rescued by helicopter and hospitalized in Reno where he received many blood transfusions.

Despite the ordeal, Paul was back at work in six weeks, though he needed surgery later.

In the pictures above, Paul shows a T-shirt that commemorates the fall he suffered.

That I had a phone connection after falling so far from so high up on the mountain as a snowstorm came in? he recalled. Truly amazing! In 2004, phone technology wasnt nearly as good as it is now. I have to hand it to AT&T because getting off the mountain to critical care was essential for my survival and then excellent medical care that included blood transfusions.

Paul was a blood donor before 2004. Hes now made 110 blood donations, mostly whole blood donations because his Type O-negative blood is especially needed by trauma patients.

I have been very fortunate with my wife and family, friends, my job and life in general, he said.

Paul first shared his story with KCRA 3 for the blood drive in 2012.

Natasha Deegan contracted Hepatitis A from food poisoning in 2010 and became one of a few very who progress to the state where a liver transplant is necessary.

A one-percenter, she said with a flash of humor.

Natasha was in an induced coma prior to her liver donation and received blood transfusions throughout the experience.

She recently celebrated 10 years post-transplant and thanks blood donors for the important part they played in her medical crisis.

Natasha said she has cherished every day and lived my life to the fullest.

I have traveled extensively with my husband and celebrated my 40th birthday in Singapore and the Maldives. Years may have passed but the gratitude has never decreased, she said. Thank you to all the blood donors. I am proof you save lives."

Natasha first shared her story with KCRA 3 for the blood drive in 2014.

Bobby Karratti looked like a blueberry baby at 3 months old, according to his mom, Alex.

His skin was covered with blue pinpricks that indicated a clotting disorder.

He had no platelets and after many months of further tests and misdiagnoses, he was diagnosed with Wiskott-Aldrich syndrome (WAS), a rare and life-threatening blood disease.

Bobby needed every blood component platelets, red cells, plasma to survive. Since turning 3 months old, he was in a hospital, visiting an infusion center, receiving blood transfusions, or seeing a doctor nearly every day until he received a bone marrow transplant the only known cure on Aug. 15, 2013. It was just a few weeks before his second birthday.

Now, Bobby is healthy and celebrating eight years post-transplant. Hes a fifth-grader, loves sports, and keeps his mom and day on their toes with every new adventure. They are so grateful for blood donors who helped their son into his healthy future.

Bobby's family first shared his story with KCRA 3 for the blood drive in 2018.

Darnay McMillan was born with sickle cell disease (SCD). Her twin brother wasnt.She would go on to receive her first blood transfusion at age 6.

By 12, Darnay was receiving transfusions each month to help manage her condition.

We dont know where we would be without blood donors, said Darnays mom, Maya. Words cant express how I feel but blood donors are a blessing to our family.

When the disease was managed well Darnay didnt miss school. As she got older, she started learning more about how a bone marrow transplant could help her, though her mom wasnt enthusiastic about the risks.

I knew that sickle cell disease could shorten my life, Darnay said. Even though the idea of a transplant was scary, the opportunity of a healthier life was enough to convince me to try.

When she was age 15 she convinced her mom that she was ready to go ahead with the marrow transplant and received it from an unrelated donor in 2015.

It wasnt easy. Darnay struggled with graft vs. host disease, a condition where new marrow donor cells can attack the host. But that improved over time.

Darnay graduated from high school with her friends and went on to college before the pandemic closed things down.

Now age 20, her health at six years post-transplant is good and she remains grateful to blood donors.Blood donors even though I dont know them personally are part of my history, she said. With their gifts, I was healthy enough to get a bone marrow transplant that cured me of sickle cell. I was able to graduate high school, go on to college, meet new people and experience adulthood.

Darnay and her family first shared their story with KCRA 3 for the blood drive in 2013.

Healthy, smart and competitive on the volleyball court, Ashley Vanderpan had already been recruited by Syracuse University and looked forward to her last years of high school.

But in the fall of 2016 when she was a sophomore, a nagging cough developed into severe breathing problems then hospitalization.

Ashley was put into a medically-induced coma on Dec. 13, 2016, and was not revived until two months later.

Her need for blood products included about 300 blood components, plasma, red blood cells and platelets about 37 gallons.

I didnt even realize you could give blood components like plasma and platelets, her father Dave said. Every few days Ashley would receive so much blood that I saw how necessary it was to have blood products on hand.

Ashleys top goal was to get back on the volleyball court, first by walking (with a medical port in her chest), then adding distance and volleyball drills.

The single-mindedness resulted in her achieving her goal. She was back on the court in the fall of 2017 and also became homecoming queen.

Ashley attended Syracuse University then transferred to San Diego State where she played volleyball and is now a senior looking forward to pursuing a masters degree in speech-language pathology.

She recently celebrated her 21st birthday, happy to be healthy and strong.

Im living proof of what blood donation can do, she said. I was shocked at the number of blood transfusions I needed. Hundreds of people donated for me. Its hard for me to imagine but Im so thankful and motivated to be a better person and a better volleyball player. Im hopeful that a younger generation of new donors who donated for me will continue to keep donating and help others.

Ashley and her family first shared their story with KCRA 3 for the blood drive in 2018.

See the original post:
Next Chapters: How Northern California blood recipients are doing years after donors helped save their lives - KCRA Sacramento

Fans of bodybuilding are familiar with the health struggles that have plagued eight-time Mr. Olympia Ronnie Coleman in recent years. Coleman has endured over a dozen surgeries to his neck and back and, as a result, walks with crutches. Through it all, Coleman maintains a positive spirit and remains optimistic thanks in part to two stem cell treatments, which are starting to pay off.

Coleman joined co-host Giles Thomas in a recent episode of the Aint Nothing but a Podcast show. In the video, released on Dec. 29, 2021, Coleman says that hes beginning to feel like his old self again, and his weight gain reflects his health improvements.

Sporting a Ronnie Coleman Signature Series shirt that showed off his noticeable arm improvement, the Texas native his home revealed that hes back up to 285 pounds. And if youre having trouble believing that Coleman weighs close to what he did in his competitive prime, youre not alone.

I weighed myself five times on the scale downstairs, and I thought maybe its because Im downstairs,' Coleman said on the podcast episode. I was freaking out, so I went upstairs, and [that scale] was the same. I was like wow.'

BarBend reached out to the 2016 International Sports Hall of Fame inductee directly to follow up on what he revealed on his podcast. Coleman was happy to share more about his progress, including what he considers the best improvement of all the return of his signature leg size.

Thats the thing Im most proud of, Coleman tells BarBend. My legs had atrophied a whole lot since 2016 when I went in and had my first surgery 2017, same thing, 2018, 2019, 2020, same thing. I was just about to give up on it, you know. Then, suddenly, about four months ago, I started feeling a pump in my legs. And then I noticed the size had come back, and the atrophy was gone. I was geeked!

The man considered the most legendary bodybuilder of all time wasnt just known for being big. His freakish feats of strength including an 800-pound back squat and deadlift only added to the mystique he brought to the stage.

While Coleman isnt going to be moving that kind of weight anytime soon, hes been more active on social media, sharing training clips, such as the one below in which he performed a set of leg extensions on Dec. 12, 2021.

Coleman isnt leg-pressing 2,300 pounds as he did in his prime, but he is throwing more 45s on the machine nowadays than during his recovery.

Im back up to doing five plates, one each side, up from three per side a few months ago, Coleman says.

The eight-time Mr. Olympias improvements arent exclusively in the lower body. Coleman shared that hes getting stronger on numerous lifts in the gym. For example, Coleman is moving weight, performing 20 reps of rear lateral raises. The new size is evident, and his trademark smile was on full display during the set (see below).

My strength has come up a whole lot. Im going to say that its up about 40 percent.

He used the flat dumbbell press as another example, saying that he is now working with 70-pound dumbbells for his sets of 20 reps, which he does for every lift. He is training six days a week, as he did during his reign as the number one bodybuilder on the planet.

Returning leg strength is undoubtedly a strong sign that Colemans physical health is improving. That said, the former police officer mentions that itll still be a while before hes able to ditch the crutches.

My feet are still numb, and my quads are still numb, but theyre not quite as numb. I can start to feel them a little bit, he says. Once Im able to relieve this numbness, I will stand a much better chance of balancing myself.

Coleman says that the stem cell specialist told him that nerve regeneration takes about two years. As Coleman explains on the podcast, the specialists claim was verified when the numbness in his neck went away after two years, almost to the day. Coleman is confident that the same will happen with his lower extremities.

I thought about what the doctor said, and he was right. Thats what Im looking at now. It will be about two years before I get my full mobility and balance back. Then I can work on walking unassisted.

With his most recent stem cell treatment having taken place on Dec. 27, 2021, Coleman is optimistic that hell keep on progressing. This positive news caps off a good year overall for the 57-year-old icon.

In September, he was honored with the Arnold Classic Lifetime Achievement award by fellow Mr. Olympia Arnold Schwarzenegger. As great as that honor was, hes even more excited to get back out to events and meet fans now that he is in better shape and spirits I cant wait!

Featured Image: @ronniecoleman8 on Instagram

Originally posted here:
Exclusive: Ronnie Coleman on Recent Weight Gain, Current Strength, and Health Progress - BarBend

Updates from OTL-201 Clinical Proof-of-Concept Study in MPS-IIIA and OTL-204 Preclinical Study for GRN-FTD at ESGCT Showcase Potential for HSC Gene Therapy in Multiple Neurodegenerative Disorders

Launch Activities for Libmeldy Across Key European Countries, including Reimbursement Discussions, Progressing in Anticipation of Treating Commercial Patients

Frank Thomas, President and Chief Operating Officer, to Step Down Following Transition in 2022; Search for a Chief Financial Officer Initiated

Cash and Investments of Approximately $254M Provide Runway into First Half 2023

BOSTONandLONDON, Nov. 04, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today reported financial results for the quarter ended September 30, 2021, as well as recent business updates and upcoming milestones.

This quarter, we are pleased by the progress demonstrated by our investigational neurometabolic HSC gene therapy programs with promising preclinical and clinical updates at ESGCT, said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. With follow-up in OTL-201 for MPS-IIIA patients now ranging between 6 and 12 months, biomarker data remain highly encouraging, showing supraphysiological enzyme activity and corresponding substrate reductions in the CSF and urine. The launch strategy for Libmeldy is also advancing in Europe with momentum building on reimbursement discussions and patient finding activities.

Recent Presentations and Business Updates

Data presentations at ESGCT

Clinical and pre-clinical data from across the companys investigational hematopoietic stem cell (HSC) gene therapy portfolio were featured in two oral and seven poster presentations at the European Society of Gene & Cell Therapy Congress (ESGCT) on October 19-22. Highlights from key presentations are summarized below:

OTL-201 for Mucopolysaccharidosis type IIIA (MPS-IIIA): A poster presentation featured supportive biomarker data from the first four patients with evaluable results, with duration of follow-up ranging from 6 to 12 months. The treatment has been generally well-tolerated in all enrolled patients (n=5) with no treatment-related serious adverse events (SAEs). Supraphysiological N-sulphoglucosamine sulphohydrolase ( SGSH) enzyme activity above the normal range was seen in leukocytes and plasma within one to three months in all evaluable patients (n=4).A greater than 90% reduction in urinary glycosaminoglycans (GAGs) was seen within three months in all evaluable patients (n=4).SGSH activity in the cerebrospinal fluid (CSF) increased from undetectable at baseline to within or above the normal range by six months in all patients with available data (n=3).CSF GAGs decreased from baseline in patients with available data (n=3).OTL-204 for Progranulin-mutated Frontotemporal Dementia (GRN-FTD): Preliminary in vivo data from the preclinical proof-of-concept study showed that murine GRN -/- HSPCs, transduced with an LV expressing progranulin under the control of a novel promoter, are able to engraft and repopulate the brain myeloid compartment of FTD mice and to locally deliver the GRN enzyme.

R&D Investor Event Summary

In September, Orchard hosted an R&D investor event highlighting its discovery and research engine in HSC gene therapy, including an update on the OTL-104 program in development for NOD2 Crohns disease (NOD2-CD) and potential new applications in HSC-generated antigen-specific regulatory T-cells (Tregs) and HSC-vectorization of monoclonal antibodies (mAbs).

The discussion also covered the differentiated profile of Orchards HSC gene therapy approach, which has exhibited favorable safety, long-term durability and broad treatment applicability.

In particular, Orchards lentiviral vector-based HSC gene therapy programs have shown no indication of insertional oncogenesis and no evidence of clonal dominance due to integration into oncogenes. Importantly, the promoters and regulatory elements of Orchard vectors are derived from human (not viral) sequences and are specifically designed to have limited enhancer activity on neighboring genes thereby mitigating the potential for safety concerns.In addition, because of the fundamental biological differences between the HSC and adeno-associated virus (AAV) gene therapy approaches, Orchards programs have not, to date, seen the safety and durability concerns experienced by the AAV gene therapy field.

Libmeldy (atidarsagene autotemcel) launch in Europe

Orchard is providing an update on the following key launch activities for Libmeldy in Europe:

Discussions with health authorities and payors are underway across Europe in key markets including Germany, the UK, France and Italy.Qualification of treatment centers is progressing with The University of Tbingen in Germany ready to treat commercial patients and other centers in the final stages of qualification and treatment readiness.Disease awareness and patient identification activities continue and have supported patient referrals in major European centers. Orchards partnerships in the Middle East and Turkey allow for opportunities to treat eligible patients from these territories at qualified European centers.Orchard is providing sponsorship for an ongoing newborn screening pilot in Germany and is working with laboratories to implement pilots in Italy, the UK, France and Spain.

Executive organizational update

The company also announced that Frank Thomas will step down from his role as president and chief operating officer, following a transition in 2022. A search for a chief financial officer is underway. Mr. Thomas other responsibilities will be assumed by existing members of the leadership team in commercial and corporate affairs. Orchard recently strengthened the executive team with the appointments of Nicoletta Loggia as chief technical officer and Fulvio Mavilio as chief scientific officer and the promotion of Leslie Meltzer to chief medical officer.

I want to extend my gratitude to Frank Thomas for his immense contributions to Orchard, said Gaspar. During his tenure, Frank oversaw the transition of the organization to a publicly traded company and has managed operations with a focus on cross-company innovation, including his role as a key architect in creating and executing the focused business plan we rolled out in 2020. Along with the entire board of directors and leadership team, I appreciate Franks commitment to facilitate a smooth transition during this time.

Gaspar continued, Our search is focused on a CFO to lead the broad strategic planning efforts necessary to capitalize on the full potential of our hematopoietic stem cell gene therapy platform. We have a strong team in place to aid Orchards success in this next phase of growth and are well capitalized through the anticipated completion of several value-creating milestones.

Upcoming Milestones

In June 2021, Orchard announced several portfolio updates following recent regulatory interactions for the companys investigational programs in metachromatic leukodystrophy (MLD), Mucopolysaccharidosis type I Hurler syndrome (MPS-IH) and Wiskott-Aldrich syndrome (WAS).

OTL-200 for MLD in the U.S: Based on feedback received from the U.S. Food and Drug Administration (FDA), the company is preparing for a Biologics License Application (BLA) filing for OTL-200 in pre-symptomatic, early-onset MLD in late 2022 or early 2023, using data from existing OTL-200 patients. This approach and timeline are subject to the successful completion of activities remaining in advance of an expected pre-BLA meeting with FDA, including future CMC regulatory interactions and demonstration of the natural history data as a representative comparator for the treated population.OTL-203 for MPS-IH: Orchard is incorporating feedback from FDA and the European Medicines Agency (EMA) into a revised global registrational study protocol, with study initiation expected to occur in 2022.OTL-201 for MPS-IIIA: Additional interim data from this proof-of-concept study are expected to be presented at medical meetings in 2022, including early clinical outcomes of cognitive function.OTL-103 for WAS: The company expects a MAA submission with EMA for OTL-103 in WAS in 2022, subject to the completion of work remaining on potency assay validation and further dialogue with EMA. The company will provide updated guidance for a BLA submission in the U.S. following additional FDA regulatory interactions.

Third Quarter 2021 Financial Results

Revenue from product sales of Strimvelis were $0.7 million for the third quarter of 2021 compared to $2.0 million in the same period in 2020, and cost of product sales were $0.2 million for the third quarter of 2021 compared to $0.7 million in the same period in 2020. Collaboration revenue was $0.5 million for the third quarter of 2021, resulting from the collaboration with Pharming Group N.V. entered into in July 2021. This revenue represents expected reimbursements for preclinical studies and a portion of the $17.5 million upfront consideration received by Orchard under the collaboration, which will be amortized over the expected duration of the agreement.

Research and development (R&D) expenses were $20.8 million for the third quarter of 2021, compared to $14.7 million in the same period in 2020. The increase was primarily due to higher manufacturing and process development costs for the companys neurometabolic programs and lower R&D tax credits as compared to the same period in 2020. R&D expenses include the costs of clinical trials and preclinical work on the companys portfolio of investigational gene therapies, as well as costs related to regulatory, manufacturing, license fees and development milestone payments under the companys agreements with third parties, and personnel costs to support these activities.

Selling, general and administrative (SG&A) expenses were $13.0 million for the third quarter of 2021, compared to $13.0 million in the same period in 2020. SG&A expenses are expected to increase in future periods as the company builds out its commercial infrastructure globally to support additional product launches following regulatory approvals.

Net loss was $36.4 million for the third quarter of 2021, compared to $20.3 million in the same period in 2020. The increase in net loss as compared to the prior year was primarily due to higher R&D expenses as well as the impact of foreign currency transaction gains and losses. The company had approximately 125.5 million ordinary shares outstanding as of September 30, 2021.

Cash, cash equivalents and investments as of September 30, 2021, were $254.1 million compared to $191.9 million as of December 31, 2020. The increase was primarily driven by net proceeds of $143.6 million from the February 2021 private placement and $17.5 million in upfront payments from the July 2021 collaboration with Pharming Group N.V., offset by cash used for operating activities and capital expenditures. The company expects that its cash, cash equivalents and investments as of September 30, 2021 will support its currently anticipated operating expenses and capital expenditure requirements into the first half of 2023. This cash runway excludes an additional $67 million that could become available under the companys credit facility and any non-dilutive capital received from potential future partnerships or priority review vouchers granted by the FDA following future U.S. approvals.

About Libmeldy / OTL-200 Libmeldy (atidarsagene autotemcel), also known as OTL-200, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD. The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability. For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the EMA website. Libmeldy is approved in the European Union, UK, Iceland, Liechtenstein and Norway. OTL-200 is an investigational therapy in the US.

Libmeldy was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. About Orchard

At Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases. We aim to do this by discovering, developing and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patients own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease in a single treatment.

In 2018, the company acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Today, Orchard has a deep pipeline spanning pre-clinical, clinical and commercial stage HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist.

Orchard has its global headquarters inLondonandU.S. headquarters inBoston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website ( http://www.orchard-tx.com ), the investor relations website ( ir.orchard-tx.com ), and on social media ( Twitter and LinkedIn ), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, including its plans and expectations for the commercialization of Libmeldy, the therapeutic potential of Libmeldy (OTL-200) and Orchards product candidates, including the product candidates referred to in this release, Orchards expectations regarding its ongoing preclinical and clinical trials, including the timing of enrollment for clinical trials and release of additional preclinical and clinical data, the likelihood that data from clinical trials will be positive and support further clinical development and regulatory approval of Orchard's product candidates, and Orchards financial condition and cash runway into the first half of 2023. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the risk that prior results, such as signals of safety, activity or durability of effect, observed from clinical trials of Libmeldy will not continue or be repeated in our ongoing or planned clinical trials of Libmeldy, will be insufficient to support regulatory submissions or marketing approval in the US or to maintain marketing approval in the EU, or that long-term adverse safety findings may be discovered; the risk that any one or more of Orchards product candidates, including the product candidates referred to in this release, will not be approved, successfully developed or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates; the delay of any of Orchards regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates or the receipt of restricted marketing approvals; the inability or risk of delays in Orchards ability to commercialize its product candidates, if approved, or Libmeldy, including the risk that Orchard may not secure adequate pricing or reimbursement to support continued development or commercialization of Libmeldy; the risk that the market opportunity for Libmeldy, or any of Orchards product candidates, may be lower than estimated; and the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter endedSeptember 30, 2021, as filed with theU.S. Securities and Exchange Commission(SEC), as well as subsequent filings and reports filed with theSEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

Investors Renee Leck Director, Investor Relations +1 862-242-0764 Renee.Leck@orchard-tx.com

Media Benjamin Navon Director, Corporate Communications +1 857-248-9454 Benjamin.Navon@orchard-tx.com

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Orchard Therapeutics Reports Third Quarter 2021 Financial Results and Highlights Recent ... - KULR-TV

Phovia is highly effective for treating superficial and deep skin infections.

This article is sponsored by Vetoquinol.

Superficial bacterial folliculitis, also called superficial pyoderma, is a commonly diagnosed dermatological condition in dogs.1,2 These infections are secondary to primary conditions affecting normal skin barrier function (eg, allergic skin disease, trauma, burns), keratinization (eg, nutritional deficiency, liver disease), and immune regulation (eg, neoplasia, autoimmunity, endocrinopathy).2 Cats less commonly develop superficial pyoderma perhaps because of decreased adhesion of staphylococci to feline corneocytes, but the primary issues causing infection are similar to those seen in dogs.3-8

The primary pathogen associated with superficial pyoderma in dogs and cats is a normal resident of the skin, Staphylococcus pseudintermedius, but other flora may be involved.2,8-12 As the normal homeostasis of this organism is disrupted from a primary disease, these gram-positive cocci invade deeper regions of the epidermis and hair follicle epithelium, increase in number, and enhance inflammation.

Classical clinical lesions of superficial pyoderma include papules and pustules that may eventually progress to alopecia, epidermal collarettes, scales, and crusts. Often the skin is erythematous and pruritic. Chronic cases may demonstrate lichenification, hyperpigmentation, and scarring alopecia from long-standing inflammation and infection.2 Cats may develop even more unique cutaneous reaction patterns and skin lesionsespecially when allergic skin disease is presentincluding miliary dermatitis, eosinophilic plaques, rodent ulcers, and eosinophilic granulomas.5

Identifying and addressing the primary disease is paramount in achieving complete, permanent resolution of the superficial pyoderma. Therefore, treatment is multifactorial and aimed at addressing the primary disease, reducing skin inflammation, and treating the infection directly. Current guidelines for the treatment of superficial pyoderma in dogs recommend the use of topical antimicrobials as sole therapy whenever possible; however, overuse of systemic antibiotics remains common.2,13-16

Topical therapy has many benefits including direct antimicrobial effects without use of an antibiotic, reduction in antibiotic-resistant bacterial populations, restoration of the normal skin barrier, enhancement of skin hydration, physical removal of keratinous debris, and removal of offending allergens from the haircoat.2,14 However, topical therapy is met with challenges that impede clinical application. Adherence is the biggest concern when recommending topical therapy to pet owners. Frequent bathing or application of medicated solutions to the skin can be difficult when busy owner lifestyles combine with a nonadherent patient. Skin inflammation can be painful and animals may be resistant to topical therapy. Cats are fastidious groomers and may lick away a medicated topical therapy before it can achieve appropriate contact time. Additionally, some topical agents can cause oral erosions and ulcerations or even gastrointestinal disturbance when groomed off. For these reasons, systemic antibiotics continue to be a common prescribing practice for superficial pyoderma.

All antibiotic use, despite duration or frequency, contributes to the development of antibiotic-resistant bacterial populations on the animal and in the environment.17-19 From that very first dose, bacteria are constantly evolving to implement inherent and acquired resistance mechanisms necessary for survival. One well-recognized mechanism is oxacillin resistance through the mecA gene, which produces a penicillin-binding protein receptor with poor affinity for -lactam antibiotics.2,14,15,20-23 Even more concerning than these oxacillin-resistant strains are those that develop multidrug resistance, which is defined as resistance to 3 or more antibiotic drug classes. This may happen over time with repeated antibiotic exposure or after a single dose of certain antibiotics such as fluorinated quinolones.2,20,23-25 The continued emergence of antibiotic-resistant bacteria inhibits the successful treatment of bacterial infections in pets and humans. As veterinarians consider how their antibiotic use contributes to this growing pandemic, they must look for alternative, safe, effective, affordable, and convenient antibacterial treatment modalities.

Phovia as a solution

Investigation into the photobiological effects of light therapy has been ongoing for the past 50 years. Photobiomodulation (PBM) therapy is a type of light treatment that uses visible or near infrared light to promote therapeutic benefits including induction of tissue healing and regeneration and inhibition of biological responses that induce pain or inflammation. The treatment distance, wavelength, fluence, pulse parameters, spot size, and irradiation time influence the effects of light energy on tissue. Visible light with wavelengths ranging from 400 to 700 nm can stimulate positive photobiomodulatory effects that promote wound healing, reduce inflammation and pain, modulate stem cell populations, and reduce bacterial contamination of wounds.26,27

Once visible light enters the skin, it is absorbed by the cells and initiates chemical changes dependent on the wavelength (or color) of light and the chromophore within the skin.27 Within each cell, membrane-bound organelles called mitochondria contain chromophores that absorb the light energy and begin making energy (adenosine triphosphate; ATP) via activation of cytochrome c oxidase. Outcomes of the mitochondrial respiratory pathway activation include stimulation of secondary messenger pathways, production of transcription factors and growth factors, and increased ATP production. However, excessive light energy exposure will overstimulate mitochondrial respiration and cause expenditure of all ATP reserves, which creates oxidative stress resulting in damaging elevations of nitric oxide, production of harmful free radicals, and activation of cytotoxic mitochondrial-signaling pathways leading to apoptosis.27,28 This is why creating PBM therapy protocols is important for targeting the beneficial effects while avoiding unintended harm.

Specific benefits of light energy within the visible light spectrum can be broken down into each color of light. Blue light (400-500 nm) has a lower penetration depth and primarily interacts with keratinocytes, reduces bacterial adhesion and growth, and increases intracellular calcium and osteoblast differentiation.29-31 Green light (495-570 nm) affects the superficial tissue and alters melanogenesis, reduces hyperpigmentation of the skin, and reduces tissue swelling.29,30 Red light (600-750 nm) penetrates deeper into the dermis and subcutis where it acts on cellular mitochondria to reduce inflammation and promote collagen synthesis through fibroblast proliferation and production of transforming growth factor-, fibroblast growth factor, platelet derived growth factor, and others.26-28,32,33 Red light has proliferative effects on mesenchymal stem cells and induces proliferation of epithelial colony forming units important for tissue repair and regeneration.34,35

Phovia, sold by Vetoquinol, is a form of fluorescent PBM therapy utilizing a blue light emitting diode (LED lamp, 400-460 nm) and topical photoconverter gel that emits low-energy fluorescent light (510-600 nm) when illuminated by the LED lamp.36,37 This interaction results in the formation of multiple wavelengths of visible light, each with a unique depth of penetration and effect on the tissue as described above. Application is fast and simple. The affected skin may be clipped free of hair and cellular debris removed with gentle cleaning. The skin is allowed to dry before application of the photoconverter gel. Just prior to application, 1 ampule of fluorescence chromophore gel is added to 1 container of photoconverter carrier gel and mixed thoroughly. The mixture is applied in a 2-mm layer to the affected skin, and the LED lamp is held 5 cm above the lesion and used to illuminate the area for 2 minutes. The gel is wiped away using saline-soaked gauze. The application can be repeated immediately after 5 to 10 minutes of rest or a second application can occur a few days later. Twice-weekly applications are continued until the wound is healed. Appropriate eyewear is required to protect the operator from the intensely bright light. Application is pain free and stress free for the patient, so sedation is not typically required.

Benefits of Phovia

Phovia shows great promise as a safe, effective therapy for treatment of numerous inflammatory dermatoses in dogs including superficial pyoderma,38 deep pyoderma,39 perianal fistula,40 interdigital dermatitis,41 calcinosis cutis,42 acute traumatic wounds,43 chronic wounds,37 surgical wounds,44 and otitis externa.45 Phovia as a sole therapy speeds time to healing by 36% in canine superficial pyoderma as compared with dogs receiving oral antibiotics alone.38 In one study, dogs with superficial pyoderma were treated with Phovia alone or with an oral antibiotic alone. Dogs treated twice weekly with Phovia demonstrated complete clinical healing in about 2.3 weeks (P < .05)whereas dogs receiving oral antibiotic healed in about 3.75 weeks.38 Additionally, Phovia speeds time to healing by nearly 50% in deep pyoderma when used with an oral antibiotic (5.7 weeks of treatment) compared with dogs receiving only oral antibiotic (11.7 weeks of treatment).39 The ability of this fluorescent PBM therapy to eliminate or significantly reduce duration of exposure to antibiotics will decrease the spread of antibiotic-resistant bacterial strains within pets and humans.

Phovias high safety profile makes it a beneficial tool to implement in everyday practice. Training the veterinary team to communicate therapy benefits with clients as well as to perform treatments is fast and easy. Training the veterinary technicians to perform treatments will give the veterinarian time to examine other patients. A single back-to-back application takes about 15 minutes, so pet owners can be in and out of the clinic quickly; however, the 2 weekly treatments can be separated by a few days if the veterinarian prefers to evaluate the patient more frequently. Additionally, when used as a sole therapy, clients are not required to administer oral or topical medications at home. This greatly improves treatment adherence and success. Instruct clients to use once-daily smartphone photos to document improvement at home. This can be useful when deciding how many treatments to perform. Most cases of superficial pyoderma will resolve completely by the third treatment.38 It is a good idea to communicate to clients that 3 to 4 weekly treatments may be required.

Conclusion

Phovia is a versatile, innovative therapeutic approach to numerous types of dermatitis.36 It is easy to implement in general practice, and is safe, pain free, and affordable. Phovia is highly effective for superficial and deep skin infections and eliminates the need for clients to administer numerous at-home treatments. This greatly improves the pet-owner bond and treatment outcomes by promoting adherence. Phovia accelerates time to wound healing, which decreases duration of antibiotic exposure and may reduce risk of antibiotic resistance development in these cases.2,13,36-39 Phovias efficacy against antibiotic-susceptible and antibiotic-resistant bacteria shows promise as an alternative therapeutic approach that promotes the principles of antimicrobial stewardship.36 If you are interested in purchasing this medical device for your practice, contact your Vetoquinol service representative.

Amelia G. White, DVM, MS, DACVD is an associate clinical professor of dermatology at Auburn University College of Veterinary Medicine.

REFERENCES

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Managing superficial pyoderma with light therapy - DVM 360

Did you know that every three minutes someone receives a diagnosis of a blood cancer? According to the National Foundation for Cancer Research, blood cancers account for nearly 10% of new cancer diagnoses in the United States annually. That means that approximately 178,520 people will receive a diagnosis this year most commonly with leukemia, lymphoma or myeloma. And although survival rates have increased dramatically over the past 20 years, theres still a long way to go when it comes to this patient population.

In this special issue of CURE, youll read about some of the research going on in blood cancer right now, including new treatment options for acute myeloid leukemia and a novel therapy using BK virus-specific T cells to treat BK virus-associated hemorrhagic cystitis (a painful side effect of stem cell transplants).

Dive into the issue for two patient stories, one about a three-time acute lymphoblastic leukemia survivor who now works as a nurse on the bone marrow transplant floor at Barnes-Jewish Hospital in St. Louis. This year also marks the 20th anniversary of the Food and Drug Administrations approval of Gleevec (imatinib mesylate), the tyrosine kinase inhibitor that saved Mel Mann. Who is Mel Mann? He is the longest-living survivor who was treated with the drug as a part of a clinical trial for chronic myeloid leukemia.

And because cancer is not a disease one should have to face alone, our cover story looks at the responsibilities and experiences of caregivers to patients with blood cancer. Not only will you read about what theyve gone through, but youll also find extra resources and tips for navigating the patient-caregiver relationship.

As always, thank you for reading.

For more news on cancer updates, research and education, dont forget tosubscribe to CUREs newsletters here.

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New Treatments and Tips for Dealing With Blood Cancer - Curetoday.com

BOSTON and LONDON, Oct. 13, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the acceptance of nine abstracts at the upcoming European Society of Gene & Cell Therapy Congress (ESGCT) taking place virtually from October 19-22.

Clinical and pre-clinical data from across the companys hematopoietic stem cell (HSC) gene therapy portfolio will be featured in two oral and seven poster presentations, including an update on the ongoing proof-of-concept study of OTL-201 for the treatment of Mucopolysaccharidosis type IIIA (MPS-IIIA, also known as Sanfilippo syndrome type A), pre-clinical data from OTL-204 in frontotemporal dementia (FTD), as well as proof-of-principle for longitudinal monitoring of vector integration sites using Liquid Biopsy Integration Site sequencing (LiBIS-seq).

Additionally, Orchards scientific advisory board member and clinical collaborator Alessandra Biffi, M.D., professor of pediatrics, University of Padua and chief of the Pediatric Onco-hematology Unit of Padua Hospital, will be giving an invited presentation on the HSC gene therapy landscape for the treatment of neurodegenerative disorders, which will include an overview of several of the companys investigational programs.

The presentations are listed below, and the full program is available online on the ESGCT website. All times are Central European Summer Time (CEST).

Oral Presentation Details:

Haematopoietic reconstitution dynamics of mobilized peripheral blood- and bone marrow-derived haematopoietic stem/progenitor cells after gene therapyPresenting Author: Andrea Calabria, Ph.D., San Raffaele Telethon Institute for Gene TherapyAbstract Number: OR049Date/Time: Friday, October 22, 2021 at 10:01 CEST

Longitudinal monitoring of vector integration sites in in vivo GT approaches by Liquid-Biopsy-Integration-Site-SequencingPresenting Author: Daniela Cesana, Ph.D., San Raffaele Telethon Institute for Gene TherapyAbstract Number: OR058Date/Time: Friday, October 22, 2021 at 12:46 CEST

Poster Presentation Details:

All posters will be available on demand starting October 19, 2021 on the ESGCT website.

Development of an ex vivo Gene Therapy for Frontotemporal Dementia (FTD)Presenting Author: Yuri Ciervo, Ph.D., division of pediatric Hematology,Oncology and Stem Cell Transplantation, Womans and Child Health Department, University of Padova, Padova, ItalyAbstract Number: P077

Optimized Lentiviral Transduction Process for ex vivo CD34+ Hematopoietic Stem Cell Gene Therapy Drug Product ManufacturePresenting Author: Saranya Elavazhagan, Orchard TherapeuticsAbstract Number: P271

Clinical Trial Update: Ex-vivo autologous stem cell gene therapy in MPSIIIAPresenting Author: Brian Bigger, Ph.D., University of ManchesterAbstract Number: P361

Dissecting bone remodelling mechanisms and hematopoietic stem cell gene therapy impact in Mucopolysaccharidosis type I Hurler bone defectsPresenting Author: Ludovica Santi, Ph.D., San Raffaele Telethon Institute for Gene TherapyAbstract Number: P157

Hematopoietic reconstitution and lineage commitment in HSC GT patients are influenced by the disease backgroundPresenting Author: Andrea Calabria, Ph.D., San Raffaele Telethon Institute for Gene TherapyAbstract Number: P181

Kinetics and composition of haematopoietic stem/progenitors mobilized cells upon G-CSF and Plerixafor administration in transplant donor or patients undergoing autologous gene therapyPresenting Author: Luca Basso-Ricci, San Raffaele Telethon Institute for Gene TherapyAbstract Number: P174

Role of peripheral blood circulating haematopoietic stem/progenitor cells during physiological haematopoietic maturation and after gene therapyPresenting Author: Pamela Quaranta, San Raffaele Telethon Institute for Gene TherapyAbstract Number: P186

About Orchard TherapeuticsAt Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases. We aim to do this by discovering, developing and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patients own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease in a single treatment.

In 2018, the company acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Today, Orchard has a deep pipeline spanning pre-clinical, clinical and commercial stage HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist.

Orchard has its global headquarters inLondonandU.S. headquarters inBoston. For more information, please visitwww.orchard-tx.com, and follow us onTwitterandLinkedIn.

Availability of Other Information About OrchardInvestors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (TwitterandLinkedIn), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaBenjamin NavonDirector, Corporate Communications+1 857-248-9454Benjamin.Navon@orchard-tx.com

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Orchard Therapeutics Outlines Comprehensive Presence at the - GlobeNewswire