Category Archives: Gene therapy

CAMBRIDGE, Mass., Sept. 15, 2020 /PRNewswire/ --Obsidian Therapeutics, Inc., a biotechnology company pioneering controllable cell and gene therapies, today announced that Bristol Myers Squibb (NYSE:BMY) has exercised its option to an exclusive worldwide license to a cell therapy candidate based on Obsidian's cytoDRiVE technology for the controlled expression of the immunomodulatory factor CD40L. This announcement marks the first opt-in decision by Bristol Myers Squibbsince the companies announced their collaboration to develop novel cell therapies in January 2019. Under the terms of the agreement, Obsidian is eligible to receive potential future milestone and royalty payments.

"We are very interested in exploring innovative approaches to developing engineered cell therapies, including the cytoDRiVE platform," said Rupert Vessey, D. Phil., Executive Vice President, Research and Early Development, Bristol Myers Squibb. "By controllingthe expression of armed payloads like CD40L, Obsidian's cell therapy candidates may have the potential to overcome tumor microenvironment resistance and unlock the power of cell therapy in solid tumors and other malignancies."

"This announcement marks an important milestone validating Obsidian's cytoDRiVE platform, and we look forward to continuing to work with Bristol Myers Squibbto bring powerful new immunotherapies to patients," said Paul K. Wotton, Ph.D., Chief Executive Officer of Obsidian Therapeutics. "We are also pleased with the pace with which our own pipeline programs are progressing as we continue to advance our lead controllable tumor infiltrating lymphocyte (TIL) therapy to the clinic."

About Obsidian TherapeuticsObsidian Therapeutics is a biotechnology company pioneering controllable cell and gene therapies to deliver transformative outcomes for patients with intractable diseases. Obsidian's proprietary cytoDRiVE technology provides a way to control protein degradation using FDA-approved small molecules, permitting precise control of the timing and level of protein expression. The cytoDRiVE platform can be applied to design controllable intracellular, membrane and secreted proteins for cell and gene therapies as well as other applications. The Company's initial applications focus on developing novel cell therapies for the treatment of cancer. Obsidian is headquartered in Cambridge, Mass. For more information, please visit http://www.obsidiantx.com.

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Maggie BellerRusso Partners, LLC[emailprotected]646-942-5631

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Obsidian Therapeutics Announces Bristol Myers Squibb Opt-In of cytoDRiVE Cell Therapy Candidate - PRNewswire

TEL AVIV, Israel, Sept. 15, 2020 (GLOBE NEWSWIRE) -- VBL Therapeutics (Nasdaq: VBLT) will present today a corporate overview, including an update on the OVAL pivotal study, at the H. C. Wainwright 22nd Annual Global Investment Conference being held virtually on September 14-16, 2020.

"Our OVAL trial of VB-111 in ovarian cancer continues to progress well, with over a third of the study participants already enrolled," said Dror Harats, MD, Chief Executive Officer of VBL Therapeutics. "We are pleased that the high response rate seen in our interim analysis in March, continues to be high in the total patient population to date. With blinded data becoming more mature, we currently see a good correlation between the CA-125 and RECIST responses, as well as with preliminary PFS and OS data. So far, OVAL blinded data recapitulate what we have seen in our positive Phase 2 study, which is very encouraging."

Presentation Details:

About VBLVascular Biogenics Ltd., operating asVBL Therapeutics, is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of first-in-class treatments for areas of unmet need in cancer and immune/inflammatory indications. VBL has developed three platform technologies: a gene-therapy based technology for targeting newly formed blood vessels with focus on cancer, an antibody-based technology targeting MOSPD2 for anti-inflammatory and immuno-oncology applications, and the Lecinoxoids, a family of small-molecules for immune-related indications. VBLs lead oncology product candidate, ofranergene obadenovec (VB-111), is a first-in-class, targeted anti-cancer gene-therapy agent that is being developed to treat a wide range of solid tumors. It is conveniently administered as an IV infusion once every 6-8 weeks. It has been observed to be well-tolerated in >300 cancer patients and demonstrated activity signals in a VBL-sponsored all comers Phase 1 trial as well as in three VBL-sponsored tumor-specific Phase 2 studies. Ofranergene obadenovec is currently being studied in a VBL-sponsored Phase 3 potential registration trial for platinum-resistant ovarian cancer.

Forward Looking StatementsThis press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as anticipate, believe, could, estimate, expect, goal, intend, look forward to, may, plan, potential, predict, project, should, will, would and similar expressions. These forward-looking statements may include, but are not limited to, statements regarding our programs, including VB-111, including their clinical development, therapeutic potential and clinical results. These forward-looking statements are not promises or guarantees and involve substantial risks and uncertainties. Among the factors that could cause actual results to differ materially from those described or projected herein include uncertainties associated generally with research and development, clinical trials and related regulatory reviews and approvals, the risk that historical clinical trial results may not be predictive of future trial results, the impact of the COVID-19 pandemic on our business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines and clinical results, that our financial resources do not last for as long as anticipated, and that we may not realize the expected benefits of our intellectual property protection. A further list and description of these risks, uncertainties and other risks can be found in our regulatory filings with theU.S. Securities and Exchange Commission, including in our annual report on Form 20-F for the year endedDecember 31, 2019, and subsequent filings with theSEC. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof.VBL Therapeuticsundertakes no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise.

INVESTOR CONTACT:Michael RiceLifeSci Advisors, LLC(646) 597-6979

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VBL Therapeutics to Provide an Update on the OVAL Study Today at the H. C. Wainwright 22nd Annual Global Investment Conference - GlobeNewswire

Pfizer has tipped its pipeline to deliver new products that add more than $15 billion to revenues by 2025. The anticipated pipeline contributions led Pfizer to predict it will weather the next wave of patent expirations that is set to start in 2026.

At its virtual investor day Tuesday, Pfizer predicted (PDF) that JAK1 inhibitor abrocitinib, antisense therapy vupanorsen and a 20-valent pneumococcal conjugate vaccine can generate peak annual revenues of more than $3 billion each. Pfizer also tipped its Duchenne muscular dystrophy gene therapy to rake in an excess of $2 billion and identified six other potential blockbusters.

With Pfizer tipping another five pipeline prospects to generate revenues of $500 million to $1 billion, the Big Pharma said sales of as-yet-unapproved experimental products could come to $15 billion by 2025.

ESMO Post Show: Highlights From the Virtual Conference

Cancer experts and pharma execs will break down the headline-making data from ESMO, sharing their insights and analysis around the conferences most closely watched studies. This discussion will examine how groundbreaking research unveiled over the weekend will change clinical practice and prime drugs for key new indications, and panelists will fill you in on the need-to-know takeaways from oncologys hottest fields.

The peak years of the experimental medicines likely lie beyond 2025. The timing of the anticipated peaks of those products coincides with the years in which Pfizer expects to face another patent cliff.

In 2026 and 2027, the U.S. basic product patents on Prevnar 13, Eliquis and Xtandi are set to expire, potentially exposing products that generated sales of $6.3 billion in Pfizers home market last year to off-patent competition. All told, analysts expect Pfizer to lose up to $20 billion in sales due to patent expirations starting in 2026. Pfizers internal forecast is roughly in line with analyst estimates.

After Lyrica went off patent last year, Pfizer entered a window in which its portfolio is virtually free from the loss of exclusivity through to 2026. The lull gives Pfizer a period to get some of its pipeline prospects to market and start growing their sales, leading management to predict the current slate of experimental assets will at least replace the revenues lost to off-patent rivals starting in 2026.

That prediction rests on Pfizers ability to get key pipeline products to market. Pfizer used the virtual event to claim a growing effectiveness in that regard. As of 2015, 5% of the drugs Pfizer took into the clinic went on to win approval. By 2019, that figure had increased to 9%, causing Pfizers success rate to go from well below the industry average to slightly above the performance of its peers.

Pfizer also noted an improved phase 2 success rate. In 2017, 17% of Pfizers phase 2 trials succeeded. Since then, Pfizer has averaged 46%. The success rate so far this year stands at 53%. Pfizer claims to be moving faster, too, stating it is on course to reduce development timelines by 2.5 years between 2017 and 2021. Automation of processes accounts for the biggest anticipated time saving.

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Pfizer tips pipeline to add $15B to sales in coming years - FierceBiotech

Sep. 14 /CSRwire/ - CRB is proud to announce that theAmicus TherapeuticsResearch and Gene Therapy Center facility has wonEngineering News-Record(ENR) MidAtlantics 2020 Best Projects (Award of Merit) in the Healthcarecategory.

Two panels of industry judges reviewed more than 80 projects located throughout the region, including Delaware, Maryland, Pennsylvania, Virginia, West Virginia, and the District of Columbia. Projects were scored on the ability of the project team to overcome challenges, contribution to the industry and community, project innovation, safety, and construction/designquality.

Amicus is a biopharmaceutical company that discovers and develops medicines for rare diseases. Its global research and gene therapy center of excellence consolidates Amicus five research departments into a unified space on the top two floors of a 14-story tower. The research workplace is the convergence of technical laboratories, office, and amenity spaces. The design creates spaces that connect science, people andpurpose.

Our team provided architectural, engineering, and lab design to realize our clients vision: a standout research facility that would attract and retain top researchers, promote collaboration, and break down barriers between employees to rapidly advance these potentially life-saving therapies through the drugpipeline.

To read more about the ENR 2020 Best Projects,click here.

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Amicus Therapeutics Wins 2020 ENR Regional Award for Best Project - CSRwire.com

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The stock of drug giant Pfizer has underperformed the market in the past five years, rising just 17%, compared with the 70% gain of the S&P 500. Last month, the stock was dropped from the Dow Jones Industrial Average.

On Monday, Pfizer (ticker: PFE) began a two-day investor presentation to show how it will become a growth stock.

Chief Executive Albert Bourla and a dozen colleagues described a pipeline of vaccines and treatments that Pfizer expects will add more than $15 billion in annual revenue by 2025. Growing sales of existing products should add an additional $8 billion in annual revenuelifting revenue from an expected $41.6 billion this year to $64.6 billion in 2025.

After the coming spinoff of Pfizers slower-selling products in its Upjohn portfolio, the company expects to show top-line growth of 6% a year, Bourla said in his afternoon remarks, while delivering double-digit growth in earnings per share. On top of higher earnings numbers, Pfizer should then merit a multiple above the 13-times earnings that its stock now gets.

Investors seemed pleased with what they heard. While the S&P was up 1% in Monday trading, Pfizer stock rose 2.6% to close at $37.01.

By 2025, Pfizers hoping for approval of 6 diabetes drugs, 6 vaccines, 12 autoimmune treatments, 3 gene therapies and 14 cancer treatments.

Among the treatments that could reach annual sales above $3 billion each are: a new vaccine for pneumococcal infections; a treatment for dangerous levels of blood triglycerides; and an oral JAK-inhibitor for autoimmune disorders like arthritis.

A gene therapy for muscular dystrophywhich would compete with one under development by Sarepta Therapeutics (SRPT)could hit peak annual sales above $2 billion. A gene therapy for hemophilia could bring in $1 billion in sales, as could three vaccines against infections of the RSV virus, the c.difficile bacterium and a 5-in-1 vaccine for childhood diseases.

Never in the 23 years that I have been here have I seen as exciting a launch story as I see today, said Pfizers head of biopharmaceuticals, Angela Hwang.

Six new vaccines are targeted for launch by 2025, vaccine group head Nanette Cocero said, including a shot for Lyme disease. New data on the companys investigational vaccines will appear at a virtual meeting of infectious-disease doctors in the week of Oct. 21.

The companys guidance for 6% annual sales growth doesnt include anything for the highly anticipated vaccine against Covid-19 that it is testing with partner BioNTech (BNTX). Pfizer will discuss that vaccine in Tuesdays presentations.

Pfizer hopes to have a Covid vaccine this year. Other new drugs for 2020 include tanezumab, for the pain of osteoarthritis, and treatments for colorectal cancer and bladder cancer.

Write to Bill Alpert at william.alpert@barrons.csom

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Pfizer Highlights Dozens of Drugs in Its Pipeline. Heres What It Means to Growth. - Barron's

Report Ocean announces the release of Cell and Gene Therapy Market research report. As per Report Ocean, the market is expected to grow at a healthy pace in the coming years. Leading market vendors are focusing on the development of their mergers & acquisitions with the main aim of providing a broad geographical presence to multiple industries. Most players are anticipated to adopt three key business strategies to cement their position in the market, i.e. expanding product portfolio, facilitating product differentiation, and participating in mergers and acquisitions.

Industry challenges together with the latest developments in the technological developments of the Cell and Gene Therapy Market have been elaborated on in the market intelligence report. It also provides a detailed picture of the trends of the changing industry structure and the challenges that are faced by various industry participants. The report elaborates on the major challenges that the participants of the said market could possibly across the globe.

Request Free Sample Report athttps://reportocean.com/industry-verticals/sample-request?report_id=mai52690

The outbreak of Covid-19 has brought in uncertainties and disruptions for the present and future of several businesses across the globe. The pandemic has claimed both lives and livelihoods, there leaving little or no hope till a vaccine for Covid-19 arrives. However, analysts at Report Ocean make a careful and meticulous assessment of the present situation and disruptions caused by the virus in the supply chain to draw estimates, projections and avenues of growth for the Cell and Gene Therapy Market.

The objectives of this study are to define, segment, and project the size of the Cell and Gene Therapy Market based on company, product type, end user and key regions.

Competitive Landscape:

The report also includes several valuable information on the Cell and Gene Therapy Market, derived from various industrial sources. The report studies the competitive environment of the Cell and Gene Therapy Market report is based on company profiles and their efforts on increasing product value and production.

Key parameters which define the competitive landscape of the Cell and Gene Therapy Market:

Revenue and Market Share by Player

Production and Share by Player

Average Price by Player

Base Distribution, Sales Area and Product Type by Player

Concentration Rate

Manufacturing Base

Mergers & Acquisitions, Expansion

Market Segmentation:

The major factors are also being considered while studying the various market segmentation. Some of the key factors are study of demand and supply of Cell and Gene Therapy Market, common interests and market share of the global Cell and Gene Therapy Market market across various geographies.

Geographical Analysis

Geographically, this report is segmented into several key regions, with sales, revenue, market share, and Cell and Gene Therapy Market growth rate in these regions, from 2015 to 2026, covering;

North America (U.S. and Canada)

Europe (UK, Germany, France, Russia, Italy and Rest of Europe)

Asia-Pacific (China, Japan, India, Malaysia, Singapore, Philippines, Indonesia, Thailand, Vietnam)

South America (Brazil, Argentina, Mexico, and Rest of South America)

The Middle East and Africa (Saudi Arabia, United Arab Emirates, Turkey, Egypt, South Africa, Nigeria)

Some of the Major Highlights of TOC covers:

Cell and Gene Therapy Market Production, Revenue (Value), Price Trend by Type

Production and Market Share by Type

Revenue and Market Share by Type

Price by Type

Cell and Gene Therapy Market Analysis by Application

Consumption and Market Share by Application

Cell and Gene Therapy Market Production, Consumption, Export, Import by Region

Production, Consumption, Export, Import by Region

Production, Consumption, Export, Import by Country

Production, Revenue, Price and Gross Margin

Cell and Gene Therapy Market Manufacturing Analysis

Key Raw Materials Analysis

Market Concentration Rate of Raw Materials

Manufacturing Cost Analysis

Labor Cost Analysis

Manufacturing Cost Structure Analysis

Manufacturing Process Analysis of XYZ

Industrial Chain, Sourcing Strategy and Downstream Buyers

Cell and Gene Therapy Market Industrial Chain Analysis

Raw Materials Sources of Cell and Gene Therapy Market Major Players in 2019

Downstream Buyers

Market Dynamics

Market Drivers

Restraints

Opportunities

Increased Demand in Emerging Markets

Challenges

Porters Five Forces Analysis

Cell and Gene Therapy Market Forecast

Production, Revenue Forecast

Production, Consumption, Export and Import Forecast by Region

Production, Revenue and Price Forecast by Type

Consumption Forecast by Application

Note In order to provide more accurate market forecast, Cell and Gene Therapy Market report will be updated before delivery by considering the impact of COVID-19.

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COVID-19 Impact and Recovery Analysis - Cell and Gene Therapy Market 2020-2026 - The Market Chronicles

Integrated Health System is bringing cell and gene therapy to cats, dogs, and horses. Recently IHS Pets has helped a paralyzed dog with a spinal cord injury to walk again after it was treated with experimental PRP and prolotherapy. Click here to see the video.

Telomeres

Aging is the root of virtually every complex noncommunicable disease in humans and animals. Telomeres are the protective end caps on the ends of our chromosomes, they are as important for the health of both humans and our pets, and they play roles in longevity.

One of the contributing factors in the lifespan in dog breeds is telomere length. As in humans researchers have found that telomere length is a strong predictor of average life span among 15 different breeds consistent with telomeres playing a role in life span determination. Dogs lose telomeric DNA ~10-fold faster than humans, which is similar to the ratio of average life spans between these species. As such telomerase therapy may be beneficial to pets as well as their human caretakers.

Telomerase gene therapy has been shown to extend lifespan in animals, this therapy may help to increase bone mineral density, improve motor performance, improve metabolism, and improve brain function.

Follistatin

The loss of muscle mass with age is just as problematic for animals as it is to humans; in cats for instance a study showed that for each 100g loss of lean body mass increased the risk of death by 20%. This is typically accompanied by frailty, and it is a contributing factor to metabolic syndrome, diabetes, heart disease, and overall mortality.

Diet and exercise have been shown to pay key roles in keeping pets healthy, but the loss of muscle mass is unavoidable without an effective intervention. Enter follistatin: myostatin blocks muscle growth, when it is inhibited then follistatin is able to let muscles grow freely to stop them from wasting away.

Follistatin gene therapy has been shown to be safe and effective in animals, this therapy may help to protect against frailty, increase muscle density, increase strength, and increase endurance.

Klotho: The Queen of Anti-Aging Proteins

1 in 3 cats will suffer from renal disease, but these numbers are under scrutiny with some suggesting that estimate may be too conservative. Chronic kidney failure can occur gradually over months or years, and it is one of the most common conditions affecting older cats with most cases progressing over time worsening the disease.

Klotho is known to play a significant role in the development of chronic kidney disease, and researchers are now turning to its broader role in the anging process as a whole; such as induces expression with gene therapy in mice has been shown to extend lifespan by targeting many of the same pathways as caloric restriction. Blocking Klotho has been shown to cause premature aging.

Klotho also helps to protect the brain, and contributes to more differences in intelligence than any one single gene. Research from the University of California has shown it to protect the brains of mice and improve brain function within 4 hours; and this result included young mice, old mice, and those that were models of Alzheimers disease.

In addition Klotho also plays a critical role in the inflammaging process. Inflammaging is the long term result of the chronic physiological stimulation of the innate immune system which can become damaging during the aging process.

Circulating levels of Kloto decreases with age, this decrease is associated with an increased risk of age related disease. Gene therapy with Klotho has been shown to increase lifespan in animal models, and it may improve kidney function, brain function, clear damage caused by oxidative stress, and protect against cardiovascular disease.

With the remarkable progress being made in genetics, gene therapy may play increasingly prominent and transformative roles in medicine for both humans and animals due to the potential to treat diseases and congenital disorders.

Pets can be an important part of life, they calm us, make us laugh, and create a bond of unconditional love. The company does note that all therapies are experimental, they are not approved by any regulatory body, and they make no claims that outcomes will be positive or beneficial.

IHS Pets is the veterinary wing of Integrated Health Systems, BioViva Sciences exclusive partner. IHS connects with doctors and patients who are interested in the power of gene therapy to pave the way to healthy aging and longevity.

Continued here:
IHS Pets: Bringing Cell And Gene Therapy To Cats, Dogs & Horses - Anti Aging News

AHWATUKEE, AZ (3TV/CBS 5) -An Ahwatukee family is dealing with a worst-case scenario after two of their three young daughters were diagnosed with an extremely rare brain disease.

Only one of the girls is eligible for gene therapy, but the treatment is in a different country, and they need help with finances to make it possible.

Dave and Kendra Riley have their hands full with three girls: 5-year-old Eva, 2-year-old Olivia, and 5-month-old Keira.

But this past year, they noticed Olivia was starting to have trouble walking and talking.

Then the irises of her eyes started vibrating a little, and were like okay let's get her checked out, said Kendra.

In March, a neurologist diagnosed her with Metachromatic Leukodystrophy, known as MLD, a rare genetic brain disease. Her motor skills have quickly been regressing since.

Most cases of MLD at her age group, without any treatment, she'll maybe have another 2 to 4 years left, said Kendra.

There isn't a day that goes by where I don't cry somewhere at some point in time because it's just, what can we do to take the pain away? said Dave.

They take Olivia every week to a clinical study treatment in Iowa, but because both Kendra and Dave are carriers of genes that formed the mutation, they had to get their other two daughters tested.

Their oldest, Eva, is only a carrier and will live a normal life. But two weeks ago they got the results for baby Keira.

She came back with both gene mutations, so just like Olivia, she has MLD, said Kendra.

Because Keira isn't showing symptoms yet, she's eligible for a rare gene therapy treatment in Italy, where 29 kids in the world have gone through it.

Olivia will never be able to recover, but they may be able to save Keira if they can get to Italy in the next month.

The community has been raising money to help them get there amid the pandemic, and for that, the Riley's are grateful.

The smallest donations of a dollar or five dollars are just as heartwarming to us as the ones that are larger because that could be all they have, said Kendra with tears in her eyes.

Here is a link to the Riley familys GoFundMe if you would like to help donate to their expenses and the girls treatment.

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Ahwatukee family seeks treatment in Italy for daughter diagnosed with rare brain disease - AZFamily

Madison researchers have developed a safer and more efficient way to deliver a promising new method for treating cancer and liver disorders and for vaccination including a COVID-19 vaccine from Moderna Therapeutics that has advanced to clinical trials with humans.

The technology relies on inserting into cells pieces of carefully designed messenger RNA (mRNA), a strip of genetic material that human cells typically transcribe from a persons DNA in order to make useful proteins and go about their business. Problems delivering mRNA safely and intact without running afoul of the immune system have held back mRNA-based therapy, but UWMadison researchers are making tiny balls of minerals that appear to do the trick in mice.

These microparticles have pores on their surface that are on the nanometer scale that allow them to pick up and carry molecules like proteins or messenger RNA, saysWilliam Murphy, a UWMadison professor of biomedical engineering and orthopedics. They mimic something commonly seen in archaeology, when we find intact protein or DNA on a bone sample or an eggshell from thousands of years ago. The mineral components helped to stabilize those molecules for all that time.

Murphy and UWMadison collaborators used the mineral-coated microparticles (MCMs) which are 5 to 10 micrometers in diameter, about the size of a human cell in a series of experiments to deliver mRNA to cells surrounding wounds in diabetic mice. Wounds healed faster in MCM-treated mice, and cells in related experiments showed much more efficient pickup of the mRNA molecules than other delivery methods.

The researchers described their findings today in the journal Science Advances.In a healthy cell, DNA is transcribed into mRNA, and mRNA serves as the instructions the cells machinery uses to make proteins. A strip of mRNA created in a lab can be substituted into the process to tell a cell to make something new. If that something is a certain kind of antigen, a molecule that alerts the immune system to the presence of a potentially harmful virus, the mRNA has done the job of a vaccine.

The UWMadison researchers coded mRNA with instructions directing cell ribosomes to pump out a growth factor, a protein that prompts healing processes that are otherwise slow to unfold or nonexistent in the diabetic mice (and many severely diabetic people).

mRNA is short-lived in the body, though, so to deliver enough to cells typically means administering large and frequent doses in which the mRNA strands are carried by containers made of molecules called cationic polymers.

Oftentimes the cationic component is toxic. The more mRNA you deliver, the more therapeutic effect you get, but the more likely it is that youre going to see toxic effect, too. So, its a trade-off, Murphy says. What we found is when we deliver from the MCMs, we dont see that toxicity. And because MCM delivery protects the mRNA from degrading, you can get more mRNA where you want it while mitigating the toxic effects.

The new study also paired mRNA with an immune-system-inhibiting protein, to make sure the target cells didnt pick the mRNA out as a foreign object and destroy or eject it.

Successful mRNA delivery usually keeps a cell working on new instructions for about 24 hours, and the molecules they produce disperse throughout the body. Thats enough for vaccines and the antigens they produce. To keep lengthy processes like growing replacement tissue to heal skin or organs, the proteins or growth factors produced by the cells need to hang around for much longer.

What weve seen with the MCMs is, once the cells take up the mRNA and start making protein, that protein will bind right back within the MCM particle, Murphy says. Then it gets released over the course of weeks. Were basically taking something that would normally last maybe hours or even a day, and were making it last for a long time.

And because the MCMs are large enough that they dont enter the bloodstream and float away, they stay right where they are needed to keep releasing helpful therapy. In the mice, that therapeutic activity kept going for more than 20 days.

They are made of minerals similar to tooth enamel and bone, but designed to be reabsorbed by the body when theyre not useful anymore, says Murphy, whose work is supported by the Environmental Protection Agency, the National Institutes of Health and the National Science Foundation and a donation from UWMadison alums Michael and Mary Sue Shannon.

We can control their lifespan by adjusting the way theyre made, so they dissolve harmlessly when we want.

The technology behind the microparticles was patented with the help of the Wisconsin Alumni Research Foundation and is licensed to Dianomi Therapeutics, a company Murphy co-founded.

The researchers are now working on growing bone and cartilage and repairing spinal cord injuries with mRNA delivered by MCMs.

Reference: Khalil et al. (2020).Single-dose mRNA therapy via biomaterial-mediated sequestration of overexpressed proteins. Science Advances.DOI: 10.1126/sciadv.aba2422.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Safer and More Efficient Method To Deliver Gene Therapy - Technology Networks

Biogen has picked up a new, preclinical gene therapy program from Massachusetts Eye and Ear for inherited retinal degeneration due to mutations in the PRPF31 gene, among the most common causes for autosomal dominant retinitis pigmentosa. Theyre building on the work of Harvards Eric Pierce. The treatment of IRDs with highly effective AAV-based gene therapies is core to Biogens ophthalmology strategy, said Chris Henderson, the research head at Biogen. This agreement underscores our commitment to that strategy and builds off of our acquisition of Nightstar Therapeutics in 2019 and our active clinical trials of gene therapies for different genetic forms of IRD.

Sarepta has inked a collaboration with Hansa to develop their experimental drug imlifidase as a pre-treatment for their gene therapies. The drug is intended for use in patients who have neutralizing antibodies that would prevent gene therapies for Duchenne muscular dystrophy and Limb-girdle muscular dystrophy from working. Hansa gets a $10 million upfront and up to $397.5 million in milestones.

Its been raining money on Wall Street at least when it comes to drug developers. CRISPR Therapeutics $CRSP and Acceleron each raised a whopping $450 million this week after pricing follow-on offerings. CRISPR priced 6,428,572 common shares at a public offering price of $70.00 per share, while Acceleron $XLRN auctioned off 4,864,864 shares of common stock at a price to the public of $92.50 per share.

The transatlantic biotech player Immatics has completed its flip onto Nasdaq through the Arya Sciences Acquisition Corp. The cancer drug biotech will trade as $IMTX after it raised $253 million in the process. The SPAC was set up by Perceptive Advisors.

Seattle-based Neoleukin Therapeutics, meanwhile, raised $76.2 million $NLTX for its work on protein therapeutics.

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Biogen bags an AAV gene therapy program from Massachusetts Eye and Ear; Biotechs raised $1B-plus in latest round of follow-ons - Endpoints News