Category Archives: Gene therapy

DALLAS--(BUSINESS WIRE)--Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it has been added to the ICE Biotechnology Index (NYSE:ICEBIO) in accordance with the annual reconstitution of the index, effective prior to the U.S. market open on Monday, December 20, 2021.

Tayshas inclusion in this key biotechnology index provides important validation of our platform and value proposition as a company, said RA Session II, President, Founder and CEO of Taysha. We remain focused on executing our near-term clinical and regulatory milestones, which we believe will continue to increase our visibility within the investment community.

The ICE Biotechnology Index tracks the performance of qualifying U.S.-listed biotechnology companies classified within the Biotechnology Sub-Industry Group of the ICE Uniform Sector Classification schema, which is a multi-asset class industry classification taxonomy developed by ICE. The index includes companies that are engaged in the research and development of therapeutic treatments but are not focused on the commercialization and mass production of pharmaceutical drugs. The index also includes companies that are engaged in the production of tools or systems that enable biotechnology processes.

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. More information is available at http://www.tayshagtx.com.

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Taysha Gene Therapies Added to the ICE Biotechnology Index - Business Wire

RESEARCH TRIANGLE PARK The nearly 30 million Americans who suffer from rare diseases have received some good news.

The National Institutes of Health, the U.S. Food and Drug Administration, and a cadre of pharmaceutical companies and non-profit organizations have teamed up to speed the development of new gene therapy treatments. Its good news for North Carolina as well, which is home to close to 50 gene therapy and rare disease-focused businesses that provide jobs for several thousand Tar Heel residents.

Whats called theBespoke Gene Therapy Consortium or BGTC was launched a little less than two months ago. Its part of the NIHs Accelerating Medicines Partnership (AMP), a public/private collaboration to speed drug development across different diseases. Ten global pharmaceutical companies and half as many non-profit patient organizations, as well as 11 NIH institutes, centers and initiatives have signed on.

The project is managed by the Foundation for the National Institutes of Health, whose mission is to promote biomedical discoveries that improve peoples lives.

Currently there are about 7,000 rare diseases in this country, 5,000 of which are due to genetic factors, according to BGTC. A single damaged gene causes nearly 80% of rare genetic illnesses, leaving millions of patients in the U.S. to suffer without much hope of improvement. Currently, only two of these diseases have FDA-approved gene therapy treatments.

BGTC said most rare inherited diseases stem from a specific gene mutation that is already known, which makes gene therapy a promising solution.

A customized or bespoke therapy could correct or replace defective genes with functional ones, according to NIH Director Francis S. Collins, M.D., Ph.D. There are now significant opportunities to improve the complex development process for gene therapies that would accelerate scientific progress and, most importantly, provide benefit to patients by increasing the number of effective gene therapies, he said.

But development is costly, complex and time consuming. And rare disorders by definition affect only a small number of patients. So most pharma companies arent willing to invest years of research and millions of dollars to bring a single-disease gene therapy to market, said Joni L. Rutter, Ph.D., acting director of NIHs National Center for Advancing Translational Sciences.

BGTC hopes to change that paradigm. The consortium wants to start with a common gene delivery vector known as the adeno-associated virus (AAV). Its considered one of the most effective gene delivery platforms for many human diseases.

The partnership said it will support a series of research projects and clinical trials to create new tools for AAV clinical development and regulatory evaluation. The BGTC aims to make it easier, faster and less expensive to pursue bespoke therapies in order to incentivize more companies to invest in this space and bring treatments to patients, Rutter pointed out.

The goal, over time, is to find ways to cut up-front gene therapy development costs, standardize the technology, and make it available for a broader range of diseases. The BCTC program will create a universal set of analytical tests to speed up gene and vector manufacturing. And the consortium will look for ways to streamline the regulatory framework.

By leveraging on experience with a platform technology and by standardizing processes, gene therapy product development can be accelerated to allow more timely access to promising new therapies for patients who need them the most, said Peter Marks, M.D., PhD., director of the FDAs Center for Biologics Evaluation and Research.

The consortium members will contribute about $76 million over the next five years to back its projects. BGTC said it will fund research for between four and six clinical trials each focused on a different rare, single-gene disease for which no gene therapies or commercial programs currently exist. Three disease areas are targeted: Lupus, Alzheimers and Type 2 diabetes.

People with terrible genetic disorders are in dire need of solutions, Collins said. The BGTC promises to transform the field of gene therapy so we can treat, or even cure rare diseases for which no current therapy exists.

North Carolina, with its substantial biotechnology and academic resources, has become a prominent incubator for leading-edge biotechnology startups. Gene therapy is a fast-developing area within our states biotech infrastructure, said Sara Imhof, Ph.D., senior director of precision health for the North Carolina Biotechnology Center. Our related ecosystem is well positioned to contribute to and benefit from the Bespoke Gene Therapy Consortium. Its an exciting time, both for the patients who desperately need the benefits gene therapy can provide and for our innovators and industry leaders who are dedicated to this important area of science.

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Boost for NC gene therapy companies a new rare disease consortium - WRAL TechWire

In every visible way, Marley Gaskins is an average 12-year-old she enjoys painting, playing online games like Roblox with her friends and taking ukulele lessons. But until recently, her life was far from normal.

Marley was born with a one-in-a-million genetic disorder called leukocyte adhesion deficiency-1, or LAD-1, which cripples the immune system and results in recurring infections, coupled with slow wound healing.

She started getting what looked like ant bites on her skin when she turned 1, said Marleys mother, Tamara Hogue. When she was 3, she got a really big skin abscess on her stomach that landed her in the hospital for five weeks because it was just a massive infection they couldnt control. The infections, Tamara said, continued to get worse and more frequent, and Marley eventually needed round-the-clock attention.

Due to a defective gene, Marley was missing a protein that enables white blood cells to stick to blood vessel walls a crucial step these cells take before moving outside the vessel walls and into tissues to fight infections. Most kids with Marleys disorder, if untreated, die before the age of 2. The fact that she survived well past that mark without a diagnosis is remarkable in itself.

When Marley was officially diagnosed at age 8, after years of uncertainty and hospital stays, doctors in her home state of Florida said the only possible cure would be a bone marrow transplant from a matched donor. Tamara said Marleys doctors couldnt even provide a survival rate for LAD-1 patients who undergo bone marrow transplants because so few people are diagnosed with the disorder.

I just felt like there had to be another option, and that's when I started asking for a second opinion, Tamara said.

A new hope: Gene therapy at UCLA

Tamaras search for other treatment options led her to Dr. Donald Kohn, a physician-scientist and member of theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.

He was leading a new clinical trial sponsored by Rocket Pharmaceuticals and the California Institute for Regenerative Medicine, the states stem cell agency for children with LAD-1 in which doctors collect blood-forming stem cells with the defective gene from each child, add in a healthy copy of the gene in the lab and then return the corrected cells into the childs body. The therapy works by promptingthe childs body to create a continuous supply of healthy white blood cells capable of fighting infection. And because the corrected cells are the patients own, there is no risk of rejection, making the treatment far less risky than a bone marrow transplant.

By the time a patient gets a bone marrow transplant, theres a lot of infection and they may have already had lung problems or other complications, said Kohn, a distinguished professor of microbiology, immunology and molecular genetics in the UCLA College and of pediatrics and molecular and medical pharmacology at theDavid Geffen School of Medicine at UCLA. They have manyskin lesions and those tend to be the sites where the donor cells attack the recipients body in whats known as graft-versus-host disease.

To avoid the rejection risks of a bone marrow transplant, Marley became the first LAD-1 patient ever to receive the stem cell gene therapy.

I didnt hesitate in letting her be a participant in the trial because I knew in my heart that this would give her a chance at having a normal life, Tamara said.

Treatment and recovery

In July 2019, Tamara and Marley traveled from their hometown Live Oak, Florida, to UCLA. There, doctors gave the then 9-year-old Marley two medications to move the stem cells from her bone marrow into her bloodstream so that they could be collected. Her harvested stem cells were then sent to a Rocket Pharmaceuticalslab where a healthy copy of the defective gene was delivered into them.

A month later, mother and daughter returned to Los Angeles so that Marley could undergo chemotherapy. This step was necessary to clear Marleys bone marrow of the remaining defective stem cells and make space for the new transplanted ones to grow. Next, the genetically corrected stem cells were injected back into her bloodstream to replicate and make fully functioning new white blood cells.

One month after receiving the transplant, she was already feeling pretty well and her immune system was working great, Kohn said.

Last week, Kohn reported at the American Society of Hematology Annual Meeting and Exposition that Marley and five other children who received the gene therapy at UCLA,including three siblings,remain healthy and disease-free.Doctors expect that the one-time therapy will keep LAD-1 patients healthy for life.

None of the six kids have experienced new infections or inflammatory skin lesions since receiving treatment, Kohn said. He added that patients treated with the same type of gene therapy for more than a dozen other blood diseases have also shown very good recovery.

Marley and her mom went into this as the first ones ever, Kohn said. What they did was an act of bravery, so its great that weve been able to really help.

A string of firsts

More than two years out of treatment and no longer limited by the severe infections that used to keep her in the hospital for months, Marley is experiencing a lot of firsts: first time camping, first time getting her ears pierced and first time going to what she calls big school this year.

She tells me that shes thankful she has a story that makes her unique, Tamara said. Now she shares her journey even with kids at school to give others courage and hope.

For Tamara, who quit her job in banking to homeschool Marley from pre-kindergarten through fifth grade, the gene therapys success has also opened up new possibilities for her own future.

Its a rebirth for the patients and the parents, Kohn said. Its life-changing, from a life of worry to a life of health.

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UCLA gene therapy gives new life to girl born with fatal immune disorder | UCLA - UCLA Newsroom

Newswise Cancer chemotherapy has undergone a paradigm shift in recent years with traditional treatments like broad-spectrum cytotoxic agents being complemented or replaced by drugs that target specific genes believed to drive the onset and progression of the disease.

This more personalized approach to chemotherapy became possible when genomic profiling of individual patient tumors led researchers to identify specific "cancer driver genes" that, when mutated or abnormally expressed, led to the onset and development of cancer.

Different types of cancer like lung cancer versus breast cancer and, to some extent, different patients diagnosed with the same cancer type show variations in the cancer driver genes believed to be responsible for disease onset and progression. For example, the therapeutic drug Herceptin is commonly used to treat breast cancer patients when its target gene, HER-2, is found to be over-expressed, says John F. McDonald, professor in the School of Biological Sciences.

McDonald explains that, currently, the identification of potential targets for gene therapy relies almost exclusively on genomic analyses of tumors that identify cancer driver genes that are significantly over-expressed.

But in their latest study, McDonald and Bioinformatics Ph.D. student Zainab Arshad have found that another important class of genetic changes may be happening in places where scientists dont normally look: the network of gene-gene interactions associated with cancer onset and progression.

Genes and the proteins they encode do not operate in isolation from one another, McDonald says. Rather, they communicate with one another in a highly integrated network of interactions.

What I think is most remarkable about our findings is that the vast majority of changes more than 90% in the network of interactions accompanying cancer are not associated with genes displaying changes in their expression, adds Arshad, co-author of the paper. What this means is that genes playing a central role in bringing about changes in network structure associated with cancer the hub genes may be important new targets for gene therapy that can go undetected by gene expression analyses.

Their research paper Changes in gene-gene interactions associated with cancer onset and progression are largely independent of changes in gene expression is published in the journal iScience.

Mutations, expression and changes in network structure

In the study, Arshad and McDonald worked with samples of brain, thyroid, breast, lung adenocarcinoma, lung squamous cell carcinoma, skin, kidney, ovarian, and acute myeloid leukemia cancers and they noticed differences in cell network structure for each of these cancers as they progressed from early to later stages.

When early-stage cancers develop, and stayed confined to their body tissue of origin, they noted a reduction in network complexity relative to normal pre-cursor cells. Normal, healthy cells are highly differentiated, but as they transition to cancer, [T]hey go through a process of de-differentiation to a more primitive or stem cell-like state. Its known from developmental biology that as cells transition from early embryonic stem cells to highly specialized fully differentiated cells, network complexity increases. What we see in the transition from normal to early-stage cancers is a reversal of this process, McDonald explains.

McDonald says as the cancers progress to advanced stages, when they can spread or metastasize to other parts of the body, [W]e observe re-establishment of high levels of network complexity, but the genes comprising the complex networks associated with advanced cancers are quite different from those comprising the complex networks associated with the precursor normal tissues.

As cancers evolve in function, they are typically associated with changes in DNA structure, and/or with changes in the RNA expression of cancer driver genes. Our results indicate that theres an important third class of changes going on changes in gene interactions and many of these changes are not detectable if all youre looking for are changes in gene expression.

DOI:https://doi.org/10.1016/j.isci.2021.103522

Acknowledgments: This research was supported by the Mark Light Integrated Cancer Research Center Student Fellowship, the Deborah Nash Endowment Fund, and the Ovarian Cancer Institute (Atlanta). The results shown here are based upon data generated by the TCGA Research Network: http://cancergenome.nih.gov/.

About Georgia Institute of Technology

The Georgia Institute of Technology, or Georgia Tech, is a top 10 public research university developing leaders who advance technology and improve the human condition. The Institute offers business, computing, design, engineering, liberal arts, and sciences degrees. Its nearly 44,000 students representing 50 states and 149 countries, study at the main campus in Atlanta, at campuses in France and China, and through distance and online learning. As a leading technological university, Georgia Tech is an engine of economic development for Georgia, the Southeast, and the nation, conducting more than $1 billion in research annually for government, industry, and society.

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Gene Network Changes Associated with Cancer Onset and Progression Identify New Candidates for Targeted Gene Therapy - Newswise

The This research service discusses the cell and gene therapy (CGT) market and highlights some key roadblocks in viral vector manufacturing. While many CGT candidates exist in the pipeline, there is a huge capacity deficit that the industry is collaboratively trying to address.

New York, Dec. 21, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Technology Developments in Viral Vector Manufacturing for Cell and Gene Therapies" - https://www.reportlinker.com/p06192548/?utm_source=GNW

Scalability, costs, reproducibility, and overall process efficiency are some of the main pain points at each step of the viral vector manufacturing process.Many industry stakeholders are capitalizing on innovative, sustainable business models and capacity expansion investments to address shortage issues.

Biotechnology companies, such as Merck, Novartis, and Pfizer, and key contract development and manufacturing organizations, such as Thermo Fisher Scientific, Catalent, and FUJIFILM Diosynth Technologies, are investing in new capacities, expanding capacities, and developing innovative technologies to stay ahead in the CGT market. The research covers emerging technologies and trends, challenges, and opportunities across the manufacturing workflow, from upstream (viral vector production) to downstream (viral vector purification). Key developments in upstream processes for viral vector production include advanced transfection agents, novel plasmids, suspension-adapted cell culture, and stable producer cell lines. The research also discusses the general industry shift toward adopting automation, digitization, and advanced analytical processes, including on-line and in-line analytics and robust real-time analytics, to highlight the importance of analytical tools throughout the value chain. Smart technologies, such as automation and digital tools, and the adoption of artificial intelligence and big data support progress in process control and optimization while improving overall efficiencies and safety. The CGT industry works through orchestrated collaborations to develop reference standards and build process analytical technologies (PAT) to optimize manufacturing further. The research presents a birds eye view of key stakeholders and their innovative platforms and a snapshot of the collaborative ecosystem to understand the CGT industrys dynamic and fast-paced nature.Read the full report: https://www.reportlinker.com/p06192548/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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Technology Developments in Viral Vector Manufacturing for Cell and Gene Therapies - Yahoo Finance

BRAZIL - 2021/02/18: In this photo illustration a Bluebird Bio logo seen displayed on a smartphone. ... [+] (Photo Illustration by Rafael Henrique/SOPA Images/LightRocket via Getty Images)

Our theme of Gene Editing stocks remains down by about 11% year-to-date, considerably underperforming the S&P 500 which is up by a solid 23% over the same period. The theme would have actually declined by about 46% year-to-date if we exclude a single stock, Intellia Therapeutics, which is up by about 130% year-to-date. So why have gene-editing stocks lagged this year, and is a recovery looking likely in 2022? Lets take a look.

The markets have soured on high-growth and futuristic stocks amid an increasingly hawkish stance by the Federal Reserve, which is now planning as many as three interest rate hikes next year. Gene editing stocks have been hit particularly badly as they dont really generate much revenue yet. Secondly, some of the companies have also witnessed clinical setbacks or seen mixed data from their clinical trials. For example, Bluebird bio saw a big setback as some safety issues emerged in an ongoing study of a drug to treat cerebral adrenoleukodystrophy back in August. Editas Medicine also published some disappointing clinical trial results for its lead candidate, EDIT-101, which is targeted at Leber Congenital Amaurosis 10, a rare eye disorder.

So whats the outlook like for the theme? The sector is largely out of favor with the market and could see some volatility through 2022 if investors continue to move out of riskier assets amid rising interest rates. Liquidity could also be an issue for smaller players Bluebird and Editas which have been burning through cash and have seen recent clinical setbacks raising questions about whether they will ever see commercial success. That being said, the long-term upside for gene editing as a larger theme appears promising, given the potentially revolutionary drugs under development, that could cure conditions from cancer to rare genetic disorders that currently lack treatments, to more chronic conditions such as diabetes. Considering this, the theme could see upside in the long term and the recent correction could be a buying opportunity.

Below youll find our previous coverage of the Gene Editing theme where you can track our view over time.

[8/13/2021] Will Modernas Interest Boost Gene Editing Stocks?

Our indicative theme of Gene Editing stocks has returned about 11% year-to-date, compared to the S&P 500 which is up by about 19% over the same period. However, the gains have overwhelmingly come from a single stock, Intellia Therapeutics, which is up by about 3x year-to-date, after the company announced positive results from early-stage clinical trials for its experimental treatment for transthyretin amyloidosis, marking the first time genome editing was carried out inside the human body to treat disease. The five other stocks in our theme remain down year-to-date. For instance, Editas Medicine remains down by about 6.8%, while bluebird bio remains down by about 56%.

That being said, we think the outlook for gene-editing stocks is looking better. Intellias progress bodes well for the broader gene-editing space, as it validates that gene-editing technology works in humans and also that it remains safe. As more of these companies move candidates into clinical stages and provide readouts, we could see movements in stock prices across the theme. Moreover, gene-editing companies could be ripe for buyouts. For instance, Covid-19 vaccine behemoth Modernas management indicated that it was interested in expanding into other areas, including gene editing. Considering that a majority of gene-editing stocks are small to mid-cap companies, they could easily be acquired by larger players such as Moderna.

[7/1/2021] Gene Editing Stocks Are Worth A Look After Intellias Big Breakthrough

Intellia Therapeutics - a gene-editing company co-founded by CRISPR pioneer and Nobel prize winner Jennifer Doudna - indicated that NTLA-2001, its experimental treatment for transthyretin amyloidosis provided very promising results in an early state trial. Although the study was small, including just six patients, the company noted that there were significant reductions in levels of a harmful liver protein that is associated with the disease after a single infusion. Intellia stock has rallied by almost 80% over the last three trading days following the news.

Now, we think that this could be a big deal for the broader gene editing sector, as well. This was the first report from a clinical trial of genome editing carried out inside the human body to treat disease, and the results should broadly validate that gene-editing technology works in humans and also that it remains safe. Our indicative theme of Gene Editing stocks has rallied considerably over the last week, and remains up by roughly 20% year-to-date, compared to the S&P 500 which is up by about 15% over the same period. That said, the gains are primarily driven by Intellia stock, which is up by almost 3x year-to-date, and the five other stocks in our theme have actually underperformed the market, or declined this year. For example, CRISPR Therapeutics is up by just about 6%, while Vertex Pharmaceuticals and Editas Medicine are down by 15% and 19%, respectively. Sangamo Therapeutics is down 23% (chart, 10-k), while bluebird bio is down by 26%. As more of these companies move candidates into clinical stages and provide readouts, we could see gains in stock prices across the theme.

[6/14/2021] Should You Add Gene Editing Stocks To Your Portfolio?

Our indicative theme of Gene Editing stocks is down by about 12% year-to-date, compared to the S&P 500 which is up by over 13% over the same period. The decline comes as investors move money from high-growth and futuristic sectors to more cyclical and value stocks to ride the post-Covid surge in economic activity over the next few quarters. Gene Editing players have been particularly badly hit by this shift, given that they are mostly clinical or pre-clinical stage biotechs with little or no revenues. Now, although most of the companies in our theme are currently losing money, and are presently out of favor with the market, the longer-term upside could be sizable, given that they are working on potentially revolutionary drugs that could cure conditions from cancer to rare genetic disorders that currently lack treatments, to chronic conditions such as diabetes.

Within our theme, Intellia Therapeutics was the strongest performer, rising by about 57% year-to-date, due to favorable views from brokerages and anticipation surrounding the companys NTLA-2001 drug, which is a single-course, potentially curative therapy for transthyretin amyloidosis. A data readout from the phase 1 study on the drug is due later this month. On the other side, Editas Medicine has been the worst performer in our theme, declining by about -47% year to date, partly due to its big rally late last year, multiple analyst downgrades, and some changes at the top management level.

[3/29/2021] Gene Editing Stocks Have Corrected. What Next?

Our indicative theme of Gene Editing stocks is down by about 19% year-to-date, compared to the S&P 500 which is up by about 6% over the same period. With the economic recovery expected to gather pace, on the back of declining Covid-19 cases and higher vaccination rates, bond yields have been trending higher, causing investors to move funds from highly valued growth names to more cyclical and value bets. Gene Editing players have been particularly badly hit by this shift, given that they are mostly clinical or pre-clinical stage biotechs with little or no revenues. That said, we think that this could be a good time to take a look at the sector, considering that these companies are working on potentially revolutionary developments that could cure conditions from cancer to rare genetic disorders.

Within our theme, Intellia Therapeutics was the strongest performer, rising by about 19% year-to-date. Last November, the company began dosing under its phase 1 study is to evaluate its drug NTLA-2001 which is a single-course, potentially curative therapy for transthyretin amyloidosis. A data readout is due sometime in the next several months. On the other side, Editas Medicine has been the worst performer, declining by about 42% year to date, partly due to its big rally late last year, multiple analyst downgrades, and some changes at the top management level. See our earlier updates below for a detailed look at the components of our Gene Editing stocks theme.

[2/10/2021] Gene Editing Stocks To Watch

Our indicative theme of Gene Editing Stocks is up by about 187% since the end of 2018 and by about 5% year-to-date. Gene editing has received more attention this year, as scientists used the technology to cure progeria syndrome in mice, raising hopes for therapy in humans as well. Progeria is a very rare genetic condition that causes premature aging in children, shortening their lifespan to approximately 14 years. Investors also remain interested in the sector, given that it could revolutionize medicine and also due to the fact that absolute valuations arent too high, with most of the companies remaining in the mid-cap space.

Within our theme, Intellia Therapeutics (NASDAQ: NTLA) has been the strongest performer year-to-date, rising by around 35% since early January. The company recently outlined strategic priorities for 2021, which include the continued advancement of a phase 1 study for a single-course therapy for protein misfolding disorder and the planned submission of regulatory applications for the treatment of acute myeloid leukemia and hereditary angioedema this year. On the other side, Vertex Pharmaceuticals, has declined by about 10% year to date, driven partly by weaker than expected Q4 2020 results. See our updates below for a detailed look at the components in our theme.

[1/27/2021] How Are Gene Editing Stocks Faring?

Gene-editing technology is used to insert, edit, or delete a gene from an organisms genome, and shows promise in treating medical conditions ranging from cancer to rare genetic conditions. Our indicative theme on Gene Editing Stocks has returned over 170% since the end of 2018, compared to the broader S&P 500 which is up by about 54% over the same period. The theme has returned about 2.4% year-to-date. Investor interest in gene-editing remains high, given the upside potential of the sector and considering that absolute valuations arent too high, with most of the stocks remaining in the mid-cap space. Intellia Therapeutics (NASDAQ: NTLA) has been the strongest performer in our theme this year so far, rising 18% since early January. The gains come as the company has outlined strategic priorities for 2021, which include the continued advancement of a phase 1 study for a single-course therapy for protein misfolding disorder and the planned submission of a regulatory application for the treatment of acute myeloid leukemia. [1] On the other side, Editas Medicine has declined by about 13% year to date, after the company indicated that it plans to raise additional capital, issuing about 3.5 million shares at $66 per share. See our update below for a detailed look at the components in our theme.

[1/8/2021] Gene Editing Stocks

Gene editing has emerged as a promising biotech theme. The technology is used to insert, edit, or delete a gene from an organisms genome, helping to replace the defective genes responsible for a medical condition with healthy versions. This technology is being used to develop treatments for a range of diseases from cancer to rare genetic conditions, that are otherwise hard to treat, and is also being considered for diagnostic purposes. While there are broadly three gene-editing technologies, clustered regularly interspaced short palindromic repeats or CRISPR, as it is popularly known, has emerged as the method of choice with most companies, considering that it is relatively inexpensive, simpler, and more flexible compared to other tools such as ZFN and TALEN.

While most gene-editing players remain in the clinical stage with a limited financial track record, funding has risen meaningfully and larger pharma companies are also partnering with these companies, considering that the treatments could be lucrative and the broader technologies may be highly scalable. While the upside remains large, investing in these companies is risky. Being a new technology that has never been used in humans before, there are risks of significant side effects or of the therapies not being effective. The economics of producing and selling these drugs also remains uncertain. These stocks are also volatile, seeing big swings as any new research or data on their potential or risk is outlined. Our indicative theme on Gene Editing Stocks - which includes names such as CRISPR Therapeutics, Editas Medicine, and others - has returned about 230% over the past 2 years, compared to the broader S&P 500 which is up by about 52% over the same period. Below is a bit more about these companies.

CRISPR Therapeutics AG is one of the best-known names in the gene-editing space. The company is working with Vertex Pharmaceuticals to co-develop CTX001, an experimental gene therapy that has provided promising results for people with sickle cell disease, and transfusion-dependent beta-thalassemia - disorders that affect the oxygen-carrying cells in human blood. The company is also developing cancer therapy candidates independently. The company was profitable last year, due to collaboration revenues from Vertex.

CRSP

Editas Medicine, another leading CRISPR-focused biotech company, with a flagship program, EDIT-101 is targeting the treatment of hereditary blindness. The company recently finished dosing for its first group of patients in earlier-stage human trials. The company also recently filed a request with the U.S. FDA to commence phase 1/2 study of EDIT-301 in treating sickle cell disease. The company also has multiple other pre-clinical drugs focused on genetic diseases.

Intellia Therapeutics is developing a drug for a rare and fatal disease known as transthyretin amyloidosis in collaboration with Regeneron. The drug is in phase 1 trials currently. The company is also working on ex-vivo Sickle Cell Anemia treatment with Novartis that involves editing cells outside the body before infusing them into the patient. The candidate is entering Phase 1/2 trails. While the company has 8 other candidates, they are still in the research or pre-clinical stages. [2]

Sangamo BioSciences focuses on multiple areas in the genomic medicine space, including gene therapy, cell therapy, in vivo genome editing, and in vivo genome regulation. The company pioneered the zinc finger nuclease gene-editing method. The companys most advanced development is a treatment for Hemophilia A, which is being developed with Pfizer and is in phase 3 trials. The company also has 4 candidates in the phase 1/2 stage and 13 in the Preclinical stage. [3]

What if youre looking for a more balanced portfolio instead? Heres a high-quality portfolio thats beaten the market consistently since the end of 2016.

Returns Dec 2021

MTD [1] 2021

YTD [1] 2017-21

Total [2] EDIT Return -16% -57% 84% S&P 500 Return -1% 24% 107% Trefis MS Portfolio Return 0% 45% 289% [1] Month-to-date and year-to-date as of 12/22/2021

[2] Cumulative total returns since 2017

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Bluebird, Editas: Gene Editing Stocks Had A Tough Year. Will 2022 Be Better? - Forbes

Families travel to Boston Childrens Hospital from around the corner and around the globe. This year, we highlighted three of these fantastic kids.

A few months after he was born, Priyanshu was diagnosed withdouble outlet right ventricle(DORV) with hypoplastic (small) ventricle, a rare congenital heart condition. After one surgery, multiple hospitals in India declined to further treat Priyanshu due to the severity of hiscondition. Read more about how his fathers determination brought Priyanshu to Boston Childrens Benderson Family Heart Center and a lifesaving surgery.

When Laila arrived from Egypt, she had already been misdiagnosed nine times but it was no wonder. After three months at Boston Childrens, her parents learned she has trichohepatoenteric syndrome. This extremely rare genetic condition can cause chronic diarrhea, liver disease, and other complications that can be life threatening if left untreated. Learn how Lailas care team in the Congenital Enteropathy Program is helping her live her best life.

When he was just 8 weeks old, Metehan was diagnosed with type 1 spinal muscular atrophy (SMA-1), a degenerative genetic disorder that leads to loss of muscle function over time. After learning that he could receive a rare gene therapy treatment at Boston Childrens, his parents traveled for care in the hospitals Spinal Muscular Atrophy Program. Read more about how our Global Services team helped make the process as smooth as possible.

Learn more about Global Services at Boston Childrens.

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Where the world comes for answers: Meet some of our international patients Where the world comes for answers: Meet some of our international patients...

Seven people who underwent heart-bypass surgery recently in Europe volunteered to receive an additional treatment: injections of messenger RNA.

This was not one of the COVID-19 vaccines, in which the RNA code is used to teach the recipients immune system. Instead, the RNA for the surgery patients was designed to heal their hearts by promoting the growth of new blood vessels.

The study, a collaboration between drugmakers AstraZeneca and Moderna, is among dozens underway to harness the potential of RNA. Some of them started before the pandemic, but with the real-world success of the vaccines, they have now picked up steam.

At Duke University Medical Center, researchers are testing a different RNA-based drug from Moderna in patients with propionic acidemia, a rare disorder in which the liver is unable to break down certain amino acids and fats. Others are testing messenger RNA against a variety of cancers.

And, of course, RNA is being used to make more vaccines. Among those being tested are vaccines against Zika virus, respiratory syncytial virus (RSV), cytomegalovirus, and the flu.

All these efforts rely on RNAs ability to carry the recipe for proteins, the building blocks of life. In a vaccine, the protein is a harmless fragment of the virus in question, allowing the recipients immune system to practice in the event of infection. In the other drugs, the RNA can prompt patients cells to make beneficial proteins that they are unable to make themselves.

It is too soon to say how well the various non-vaccine RNA drugs will work, said cardiologist Howard J. Eisen, a medical director at the Penn State Heart and Vascular Institute, who has been following the research. Among other issues: RNA degrades quickly (remember how the COVID vaccines require cold storage?), so it has to be delivered to the right cells in a timely fashion.

Yet the potential, he says, is vast.

Itll revolutionize medicine, I think.

In the heart study, patients experienced no serious side effects as a result of the injections, the drugmakers reported in November. That was little surprise, given that billions have now been injected safely with RNA vaccines, said Eisen, who was not involved with the study.

But with just seven people (and another four who received placebo injections), the study was too small to draw conclusions about the drugs effect on heart function. Larger studies are planned.

The RNA carries the recipe for a protein called VEGF-A, a growth factor involved in forming new blood vessels. The hope is that the patients would experience an improved ejection fraction a measure of how much oxygenated blood is pumped with each heartbeat. Yet previous studies, in which researchers have sought to boost that protein with a different approach called gene therapy, have met with limited success.

Likewise, tests of the RNA-based drug for propionic acidemia are in the early stages, as are studies of RNA treatments for other metabolic diseases.

Whats clear is that new approaches for these liver disorders are sorely needed, said Dwight Koeberl, who is overseeing the Duke University site for Modernas propionic acidemia trial.

For now, patients with that disease must severely limit or avoid intake of meat, dairy, and nuts or else their bodies build up toxic byproducts that lead to neurological and heart damage, among other complications. To compensate for this restricted diet, they must drink a special formula with vitamins and other supplements. And even so, some eventually need a liver transplant.

Koeberl, a professor of pediatrics at Duke University School of Medicine, also has studied the use of gene therapy to treat such patients. That approach is a long-term fix, as the instructions for making the corrective proteins are delivered inside the nucleus of the persons cells (whereas RNA is transient, degrading within days meaning that some treatments would need to be administered multiple times).

But as with the gene therapy treatments for heart disease, gene therapy for metabolic disorders remains a work in progress. One hurdle with gene therapy is that it is typically delivered inside the recipients cells with a virus, which can be defeated by the immune system, Koeberl said.

RNA-based therapies, on the other hand, are typically packaged in tiny droplets of oily molecules called lipids, as with the COVID vaccines. These lipid nanoparticles do not enter the cell nucleus. They need to penetrate only the outer cell membrane for the RNA to fulfill its mission, and they do so with ease. Koeberl was attracted by the possibility of a more straightforward solution.

My interest is in trying to help these patients with something sooner rather than later, he said.

Many, if not most, of the RNA drugs being tested are vaccines, to judge from a search of clinicaltrials.gov, a listing of clinical studies maintained by the U.S. National Library of Medicine.

Compared to traditional vaccines, one advantage of the RNA approach is that the genetic instructions can be quickly updated to match emerging threats. Pfizer and BioNTech, for example, already are developing a vaccine to match the omicron variant of the coronavirus, though widescale production still takes time. The European Union has ordered 180 million doses of this modified vaccine, expected to be available by March.

Next-generation RNA vaccines may also have the advantage of requiring lower doses. Thats the idea behind a flu vaccine in development by Seqirus, which has U.S. operations in Summit, N.J., and is a subsidiary of CSL Limited, based in Melbourne, Australia.

The RNA in that vaccine is self-amplifying, meaning that it consists of two elements: the genetic recipe for making flu proteins that stimulate an immune response, as well as instructions to make multiple copies of that recipe. In theory, that would mean a lower dose of such a vaccine could be just as effective, yet with a lower rate of side effects. Seqirus has been studying this approach in animal models for years, and it plans to test this type of flu vaccine in human volunteers during the second half of 2022.

Patient support groups have been watching the development of messenger RNA with great interest, whether the drug is being used to prevent disease, as with the vaccines, or to treat it.

Many advocates were aware of the potential for RNA treatments long before the COVID vaccines came out. Among them is Kathy Stagni, executive director of the Organic Acidemia Association, which provides support for patients with propionic acidemia and others.

She said she has been setting the record straight every time she hears someone claim that the technology behind the COVID vaccines was rushed.

This is something theyve been working on for a long time, she said.

Eisen, the Penn State cardiologist, was working at the University of Pennsylvania decades ago when Penn scientist Katalin Karik was doing some of the early experiments that would set the stage for the vaccines.

She was not working on vaccines at the time, but on using messenger RNA to treat heart disease. Now that the technology has matured, AstraZeneca and Moderna are tackling heart disease once again.

In essence, Eisen said, it has come full circle.

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Vaccines are just the beginning for RNA. The technology is being tested on heart and liver diseases. - The Philadelphia Inquirer

DUBLIN, Dec. 21, 2021 /PRNewswire/ -- The "Global Advanced Therapy Medicinal Products CDMO Market Size, Share & Trends Analysis Report by Product (Gene Therapy, Cell Therapy, Tissue Engineered), Phase, Indication, Region, and Segment Forecasts, 2021-2028" report has been added to ResearchAndMarkets.com's offering.

The global advanced therapy medicinal products CDMO market size is expected to reach USD 12.9 billion by 2028, according to the report. It is expected to expand at a CAGR of 12.0% from 2021 to 2028.

The advanced therapy medicinal products are a group of biological products for human use that involve gene therapy products, cell therapy products, and tissue-engineered products. The growth of the market is credited to the increasing clinical trials of ATMP and the rising awareness and belief among researchers regarding the benefits of advanced therapy. The COVID-19 pandemic has significantly disrupted the cell and gene therapy industry due to the complexity in the manufacturing process.

The COVID-19 pandemic has adversely affected the overall medical industry, but the pandemic boosted the operations and development of advanced therapy due to the high requirement of the products such as mesenchymal stromal cells (MSCs) for the treatment of the virus. The regulations put forward by the FDA and government authorities have created a safe environment for the healthcare workers and allowed emergency approval for the supply of essential raw materials and faster development of the vaccines and other therapy products.

Technological advancement has been a major part of tissue engineering in the last few years. This method helps to replace or restore the injured tissues and organ function. Similarly, gene and cell therapy is attracting many patients for the treatment of rare diseases, the cases of which are augmenting globally.

Advanced Therapy Medicinal Product CDMO Market Report Highlights

Key Topics Covered:

Chapter 1 Methodology and Scope

Chapter 2 Executive Summary

Chapter 3 Advanced Therapy Medicinal Products CDMO Market: Variables, Trends, & Scope3.1 Market Segmentation and Scope3.2 Market Dynamics3.2.1 Market Driver Analysis3.2.1.1 Rising number of clinical trials for ATMP3.2.1.2 Increasing outsourcing activities3.2.1.3 Growing awareness of the treatment3.2.2 Market Restraint Analysis3.2.2.1 Stringent regulatory approvals3.2.2.2 High cost of outsourcing3.3 Penetration & Growth Prospect Mapping3.4 Advanced Therapy Medicinal Products CDMO: Market Analysis Tools3.4.1 Industry Analysis - Porter's3.4.1.1 Porter's Five Forces Analysis3.4.2 PESTEL Analysis

Chapter 4 Advanced Therapy Medicinal Products CDMO Market: Product Estimates4.1 Market Share Analysis, 2020 & 20284.2 Gene Therapy4.2.1 Gene therapy market, 2016 - 2028 (USD Billion)4.3 Cell Therapy4.3.1 Cell therapy market, 2016 - 2028 (USD Billion)4.4 Tissue Engineered4.4.1 Tissue engineered market, 2016 - 2028 (USD Billion)4.5 Others4.5.1 Market, 2016 - 2028 (USD Billion)

Chapter 5 Advanced Therapy Medicinal Products CDMO Market: Phase Estimates5.1 Market Share Analysis, 2020 & 20285.2 Phase I5.2.1 Phase I market, 2016 - 2028 (USD Billion)5.3 Phase II5.3.1 Phase II market, 2016 - 2028 (USD Billion)5.4 Phase III5.4.1 Phase III market, 2016 - 2028 (USD Billion)5.5 Phase IV5.5.1 Phase IV market, 2016 - 2028 (USD Billion)

Chapter 6 Advanced Therapy Medicinal Products CDMO Market: Indication Estimates6.1 Market Share Analysis, 2020 & 20286.2 Oncology6.2.1 Oncology market, 2016 - 2028 (USD Billion)6.3 Cardiology6.3.1 Cardiology market, 2016 - 2028 (USD Billion)6.4 Central Nervous System6.4.1 Central nervous system market, 2016 - 2028 (USD Billion)6.5 Musculoskeletal6.5.1 Musculoskeletal market, 2016 - 2028 (USD Billion)6.6 Infectious Disease6.6.1 Infectious disease market, 2016 - 2028 (USD Billion)6.7 Dermatology6.7.1 Dermatology market, 2016 - 2028 (USD Billion)6.8 Endocrine, Metabolic, Genetic6.8.1 Endocrine, metabolic, genetic market, 2016 - 2028 (USD Billion)6.9 Immunology & inflammation6.9.1 Immunology & inflammation market, 2016 - 2028 (USD Billion)6.10 Ophthalmology6.10.1 Ophthalmology market, 2016 - 2028 (USD Billion)6.11 Haematology6.11.1 Haematology market, 2016 - 2028 (USD Billion)6.12 Gastroenterology6.12.1 Gastroenterology market, 2016 - 2028 (USD Billion)6.13 Others6.13.1 Others market, 2016 - 2028 (USD Billion)

Chapter 7 Advanced Therapy Medicinal Products CDMO Market: Regional Analysis

Chapter 8 Company Profiles8.1 Strategic Framework8.2 Company Profiles8.2.1 Celonic8.2.1.1 Company Overview8.2.1.2 Financial performance8.2.1.3 Product Benchmarking8.2.1.5 Strategic Initiatives8.2.2 Bio Elpida8.2.2.1 Company Overview8.2.2.2 Financial performance8.2.2.3 Product Benchmarking8.2.2.6 Strategic Initiatives8.2.3 CGT Catapult8.2.3.1 Company Overview8.2.3.2 Financial performance8.2.3.3 Product Benchmarking8.2.3.6 Strategic Initiatives8.2.4 Rentschler Biopharma SE8.2.4.1 Company Overview8.2.4.2 Financial performance8.2.4.3 Product Benchmarking8.2.4.6 Strategic Initiatives8.2.5 AGC Biologics8.2.5.1 Company Overview8.2.5.2 Financial performance8.2.5.3 Product Benchmarking8.2.5.6 Strategic Initiatives8.2.6 Catalent8.2.6.1 Company Overview8.2.6.2 Financial performance8.2.6.3 Product Benchmarking8.2.6.6 Strategic Initiatives8.2.7 Lonza8.2.7.1 Company Overview8.2.7.2 Financial Performance8.2.7.3 Product Benchmarking8.2.7.5 Strategic Initiatives8.2.8 WuXi Advanced Therapies8.2.8.1 Company Overview8.2.8.2 Financial performance8.2.8.3 Product Benchmarking8.2.8.5 Strategic Initiatives8.2.9 BlueReg8.2.9.1 Company Overview8.2.9.2 Financial performance8.2.9.3 Product Benchmarking8.2.9.6 Strategic Initiatives8.2.10 Minaris Regenerative Medicine8.2.10.1 Company Overview8.2.10.2 Financial performance8.2.10.3 Product Benchmarking8.2.10.5 Strategic Initiatives8.2.11 Patheon8.2.11.1 Company Overview8.2.11.2 Financial performance8.2.11.3 Product Benchmarking8.2.11.5 Strategic Initiatives

For more information about this report visit https://www.researchandmarkets.com/r/rjc62f

Media Contact:

Research and Markets Laura Wood, Senior Manager [emailprotected]

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Outlook on the Advanced Therapy Medicinal Products CDMO Global Market to 2028 - Rising Number of Clinical Trials for ATMP is Driving Growth -...

Gene therapy is a powerful developing technology that has the potential to address myriad diseases. For example, Huntington's disease, a neurodegenerative disorder, is caused by a mutation in a single gene, and if researchers could go into specific cells and correct that defect, theoretically those cells could regain normal function.

A major challenge, however, has been creating the right "delivery vehicles" that can carry genes and molecules into the cells that need treatment, while avoiding the cells that do not.

Now, a team led by Caltech researchers has developed a gene-delivery system that can specifically target brain cells while avoiding the liver. This is important because a gene therapy intended to treat a disorder in the brain, for example, could also have the side effect of creating a toxic immune response in the liver, hence the desire to find delivery vehicles that only go to their intended target. The findings were shown in both mouse and marmoset models, an important step towards translating the technology into humans.

A paper describing the new findings appears in the journal Nature Neuroscience on December 9. The research was led by Viviana Gradinaru (BS '05), professor of neuroscience and biological engineering, and director of the Center for Molecular and Cellular Neuroscience.

The key to this technology is the use of adeno-associated viruses, or AAVs, which have long been considered promising candidates for use as delivery vehicles. Over millions of years of evolution, viruses have evolved efficient ways to gain access into human cells, and for decades researchers have been developing methods to harness viruses' Trojan-Horse-like abilities for human benefit.

AAVs are made up of two major components: an outer shell, called a capsid, that is built from proteins; and the genetic material encased inside the capsid. To use recombinant AAVs for gene therapy, researchers remove the virus's genetic material from the capsid and replace it with the desired cargo, such as a particular gene or coding information for small therapeutic molecules.

"Recombinant AAVs are stripped of the ability to replicate, which leaves a powerful tool that is biologically designed to gain entrance into cells," says graduate student David Goertsen, a co-first author on the paper. "We can harness that natural biology to derive specialized tools for neuroscience research and gene therapy."

The shape and composition of the capsid is a critical part of how the AAV enters into a cell. Researchers in the Gradinaru lab have been working for almost a decade on engineering AAV capsids that cross the blood-brain barrier (BBB) and to develop methods to select for and against certain traits, resulting in viral vectors more specific to certain cell types within the brain.

In the new study, the team developed BBB-crossing capsids, with one in particular AAV.CAP-B10that is efficient at getting into brain cells, specifically neurons, while avoiding many systemic targets, including liver cells. Importantly, both neuronal specificity and decreased liver targeting was shown to occur not just in mice, a common research animal, but also in laboratory marmosets.

"With these new capsids, the research community can now test multiple gene therapy strategies in rodents and marmosets and build up evidence necessary to take such strategies to the clinic," says Gradinaru. "The neuronal tropism and decreased liver targeting we were able to engineer AAV capsids for are important features that could lead to safer and more effective treatment options for brain disorders."

The development of an AAV capsid variant that works well in non-human primates is a major step towards the translation of the technology for use in humans, as previous variants of AAV capsids have been unsuccessful in non-human primates. The Gradinaru lab's systematic in vivo approach, which uses a process called directed evolution to modify AAV capsids at multiple sites has been successful in producing variants that can cross the BBBs of different strains of mice and, as shown in this study, in marmosets.

"Results from this research show that introducing diversity at multiple locations on the AAV capsid surface can increase transgene expression efficiency and neuronal specificity," says Gradinaru. "The power of AAV engineering to confer novel tropisms and tissue specificity, as we show for the brain versus the liver, has broadened potential research and pre-clinical applications that could enable new therapeutic approaches for diseases of the brain."

The paper is titled "AAV capsid variants with brain-wide transgene expression and decreased liver targeting after intravenous delivery in mouse and marmoset." Goertsen; Nicholas Flytzanis (PhD '18), the former scientific director of the CLARITY, Optogenetics and Vector Engineering Research(CLOVER)Center of Caltech's Beckman Institute; and former Caltech postdoctoral scholar Nick Goeden are co-first authors. Additional coauthors are graduate student Miguel Chuapoco, and collaborators Alexander Cummins, Yijing Chen, Yingying Fan, Qiangge Zhang, Jitendra Sharma, Yangyang Duan, Liping Wang, Guoping Feng, Yu Chen, Nancy Ip, and James Pickel.

Funding was provided by the Defense Advanced Research Projects Agency, the National Institutes of Health, and the National Sciences and Engineering Research Council of Canada.

Flytzanis, Goeden, and Gradinaru are co-founders of Capsida Biotherapeutics, a Caltech-led startup company formed to develop AAV research into therapeutics.

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New Technology is One Step Closer to Targeted Gene Therapy - Caltech