On the heels of research deals with two small biotechs, shares of Sarepta Therapeutics Inc. (NASDAQ:SRPT) hit their all-time high of more than $172 on June 23.
The share price of the Cambridge, Massachusetts-based biopharmaceutical company has doubled since mid-March. It has a market cap of more than $13 billion.
Sarepta has made a host of deals in the past several years that have made gene therapy a key part of its business, which had been dominated by drugs for muscular dystrophy. One of those pacts, with Roche (RHHBY), could be worth more than $3 billion.
Sarepta currently has a half dozen gene therapies in clinical trials, with another six waiting in the wings, according to BioPharma Dive. One of the companys compounds has shown promise in treating a type of muscular dystrophy, a genetic disease that causes weakness and wasting of the muscles in the arms and legs.
A major challenge of gene therapy is immune system reactions. Sarepta, like other developers, uses a type of virus in its therapy that, while effective, may not be able to given more than once because patients can create antibodies to it.
Thats a problem Sarepta is trying to address in its latest deals with privately held Codiak Biosciences and Selecta Biosciences Inc. (NASDAQ:SELB). In both cases, Sarepta has an option to license the biotechs' technology to develop and commercialize its therapies.
The Selecta deal focuses on gene therapies for Duchene muscular dystrophy and certain limb-girdle muscular dystrophies.
A Zion Market Research report said the global demand for the Duchenne muscular mystrophy therapeutics market was valued at approximately $2.4 billion in 2018 and is expected to grow to more than $20 billion by the end of 2025, a compound annual growth rate of more than 36% between 2019 and 2025.
Given the size of the opportunity, its no surprise Sarepta has plenty of competitors vying for a share of the business, including Pfizer Inc. (NYSE:PFE), PTC Therapeutics (NASDAQ:PTCT), FirbroGen Inc. (NASDAQ:FGEN), Roche and Bristol-Myers Squibb Co. (NYSE:BMY).
The two-year deal with Codiak gives Sarepta the right to license its technology for up to five neuromuscular diseases. If Sarepta exercises an option, Codiak will then handle research until right before the candidate goes into human testing. Sarepta will then be responsible for clinical development and commercializing the drug.
In April, the company announced it has resurrected its antiviral program in response to Covid-19. It has a therapy that is meant to block the coronavirus ability to replicate. The treatment will be tested at the U.S. Army Medical Research Institute of Infectious Diseases.
If it works, it will reduce the ability of the virus to replicate and its ability to infect other cells, Sarepta CEO Doug Ingram told Forbes. He cautioned that the drug is at an early stage.
According to CNN Money, the 21 analysts offering 12-month price forecasts for Sarepta have a median target of $192, with a high estimate of $260 and a low $152. The stock is rated a buy.
Disclosure: The author hold positions in Pfizer and Bristol-Myers Squibb.
Read more here:
Not a Premium Member of GuruFocus? Sign up for a free 7-day trial here.
Barry Cohen
He has contributed to a number of financial websites, writing primarily about the stocks of health care companies.
Read more here:
Sarepta Addressing Gene Therapy Issue With Two Acquisitions - GuruFocus.com
- Gene therapy research offers hope for people with chronic kidney disease - Medical Xpress - January 6th, 2025
- Sangamo Therapeutics to Regain Full Rights to Hemophilia A Gene Therapy Program Following Pfizers Decision to Cease Development of Giroctocogene... - January 6th, 2025
- JCR Pharmaceuticals and Modalis Therapeutics Announce Transition to the Next Phase of Joint Research Agreement for Development of Novel Gene Therapy -... - January 6th, 2025
- Gene therapy targets the retina to treat eye disease - Nature.com - January 6th, 2025
- Sangamos Stock Plummets as Pfizer Axes Hemophilia Gene Therapy Pact - BioSpace - January 6th, 2025
- How Increased Use of Gene Therapy Treatment for Sickle Cell Disease Could Affect the Federal Budget - Congressional Budget Office - January 6th, 2025
- The Future of Regulatory Processes in Cell and Gene Therapy - Pharmaceutical Executive - January 6th, 2025
- CGTLive's 2024 Pillars of Progress: Most-Watched Conference Interviews - CGTLive - January 6th, 2025
- Pfizer cuts losses on near-approval hemophilia gene therapy, adding to troubled Sangamo's woes - Fierce Biotech - January 6th, 2025
- JCR Pharmaceuticals and Modalis Advance Joint Gene Therapy Research - TipRanks - January 6th, 2025
- JCR and Modalis Advance Joint Gene Therapy Research - TipRanks - January 6th, 2025
- Novartis Gene Therapy Shows Promise in Treating SMA - Yahoo Finance - January 6th, 2025
- Gene Therapy Market to Hit Valuation of US$ 42.26 Billion By 2033 | Astute Analytica - Yahoo Finance - January 6th, 2025
- Novartis gene therapy helps children with rare muscle disorder in study - Reuters - January 6th, 2025
- Capricor Puts Rolling BLA for DMD Cardiomyopathy Cell Therapy Deramiocel in Front of the FDA - CGTLive - January 6th, 2025
- Positive data could expand use of Novartis gene therapy for SMA - Yahoo Finance - January 6th, 2025
- Sangamo spirals after Pfizer halts hemophilia A gene therapy partnership - MM+M Online - January 6th, 2025
- Cell Therapy and Gene Therapy CDMO Market to Reach USD 11.11 Billion by 2030 | Discover Growth Trends and Insights | Valuates Reports - PR Newswire - January 6th, 2025
- Struggling With Adoption, Sickle Cell Gene Therapy Manufacturers Embrace CMS Model - News & Insights - January 6th, 2025
- Sangamo Therapeutics to Regain Rights to Gene Therapy Program from Pfizer - Contract Pharma - January 6th, 2025
- Researchers Create Gene Therapy with Potential to Treat Peripheral Pain ... - December 28th, 2024
- How CRISPR Is Changing Cancer Research and Treatment - December 28th, 2024
- Gene Therapy Shows Long-Term Vision Benefits in Rare Eye Disease - December 28th, 2024
- 100 cell and gene therapy leaders to watch in 2025 - December 28th, 2024
- Can a new gene therapy reverse heart failure? - Futurity - December 28th, 2024
- Sustained visual improvements in LHON patients treated with AAV gene therapy - Medical Xpress - December 28th, 2024
- Nebraska Medicine administers novel gene therapy to first hemophilia ... - December 28th, 2024
- Gene Therapy for Cardiomyopathies Presents Promising Alternative to Current Treatment - Managed Healthcare Executive - December 28th, 2024
- Stem Cell Transplantation Still the Main Treatment Option for Beta-Thalassemia - Medpage Today - December 28th, 2024
- Caribou Overhyped Gene-Therapy Testing, Investor Class Suit Says - Bloomberg Law - December 28th, 2024
- WuXi AppTec sells off cell and gene therapy operations in US, UK - FirstWord Pharma - December 28th, 2024
- Top 5 Print Publication Articles of 2024 - Managed Healthcare Executive - December 28th, 2024
- Gene Therapy Shows Long-Term Vision Benefits in Rare Eye Disease - Medpage Today - December 28th, 2024
- UPenn gene therapy pioneers biotech gets $34 million in funding - The Philadelphia Inquirer - December 28th, 2024
- PHC Corporation to present LiCellGrow at Advanced Therapies Week 2025 - Drug Target Review - December 28th, 2024
- The Evolution of Cell & Gene Therapy: Development and Manufacturing Insights and the Role of CDMOs - Pharmaceutical Technology Magazine - December 28th, 2024
- Pig kidney transplants, new schizophrenia drug: Here are 5 of the biggest medical breakthroughs in 2024 - ABC News - December 28th, 2024
- Cell Therapy Manufacturing Trends And Advancements Continuing In 2025 - BioProcess Online - December 28th, 2024
- Can Gene Therapy Treat Chronic Pain? - LabRoots - December 28th, 2024
- Driving innovation: India's foray into gene and cell therapies - The Economic Times - December 28th, 2024
- Governor Hochul Celebrates the Opening Of New York's First Cell and Gene Therapy Hub at Roswell Park Comprehensive Cancer Center in Buffalo - PR Web - December 19th, 2024
- GenSight Biologics Provides Update on Regulatory Discussions and Financial Situation - Business Wire - December 19th, 2024
- Atsena completes dosing in part A of X-linked retinoschisis gene therapy trial - Healio - December 19th, 2024
- Astellas and Sangamo Therapeutics Announce Capsid License Agreement to Deliver Genomic Medicines for Neurological Diseases - StreetInsider.com - December 19th, 2024
- Ring Therapeutics lays off just under half of staff in 2nd wave of cuts this year, CEO set to step down - Fierce Biotech - December 19th, 2024
- Gov. Hochul celebrates opening of first cell and gene therapy hub in NYS - WIVB.com - News 4 - December 19th, 2024
- Muscular Dystrophy Association and Coalition to Cure - GlobeNewswire - December 19th, 2024
- Atsena Therapeutics Announces Dosing Completed in Part A of - GlobeNewswire - December 19th, 2024
- 'A milestone moment': Roswell Park celebrates opening New York's first cell and gene therapy hub - WKBW 7 News Buffalo - December 19th, 2024
- Gene therapy to prevent stillbirth and premature delivery developed - News-Medical.Net - December 19th, 2024
- Breaking through the blood-brain barrier - Science - December 19th, 2024
- Cell therapy weekly: partnerships for advancing cell and gene therapies - RegMedNet - December 19th, 2024
- Roswell Park Opens Cell, Gene Therapy Hub - WGRZ.com - December 19th, 2024
- Cartherics gets $300k grant to advance Cell and Gene Therapy development - ETHealthWorld - December 19th, 2024
- ELMCRx Solutions Offers Cell & Gene Therapy Support Through Partnership with Emerging Therapy Solutions (ETS) - Business Wire - December 19th, 2024
- Fueling the Future of Gene Therapies with Manufacturing Innovation, Upcoming Webinar Hosted by Xtalks - PR Web - December 19th, 2024
- Concinnity secures 3M Seed funding to advance AI-driven gene therapy safety - Tech.eu - December 19th, 2024
- Viral Vectors-Based Gene Therapy for Non-Human Primates Market to Reach Over USD 92.76 Million by 2034 - EIN News - December 19th, 2024
- The pharma industry's silence on RFK Jr., and efforts by parents to develop gene therapies for their children - STAT - December 19th, 2024
- Tenaya reports positive early data on heart gene therapy - Investing.com - December 19th, 2024
- Unraveling The Complexity Of Cell Therapy: Advancements And Challenges - Life Science Leader Magazine - November 27th, 2024
- Novartis wagers more than $1B on gene therapies for the nervous system - BioPharma Dive - November 27th, 2024
- Gene therapy for geographic atrophy in age-related macular degeneration: current insights - Nature.com - November 27th, 2024
- Novartis buys gene therapy startup Kate Therapeutics, joining pursuit of muscular dystrophy treatment - STAT - November 27th, 2024
- At MGB's gene therapy institute, effort to win first venture capital investments continues - The Business Journals - November 27th, 2024
- Neurogene reports death of Rett patient left in critical condition by high dose of gene therapy - Fierce Biotech - November 27th, 2024
- Alzheimer Disease Awareness Month 2024: Looking Back at a Year of Progress in Cell and Gene Therapy - CGTLive - November 27th, 2024
- Why This Gene-Therapy Companys Stock Is Rising 228% - Yahoo! Voices - November 27th, 2024
- How Minaris is Tackling the Scalability Challenge in Cell and Gene Therapy: A Conversation with CEO, Dr. Hiroto Bando - geneonline - November 27th, 2024
- RNA editing is the next frontier in gene therapy heres what you need to know - The Conversation - November 27th, 2024
- Assessment of gene therapy viral vectors in RPE cells - News-Medical.Net - November 27th, 2024
- Retinal organoids and RPE models for retinal gene therapy development - News-Medical.Net - November 27th, 2024
- China Vows to Bolster Gene Therapy Research in Key Biotech Hub - Bloomberg - November 27th, 2024
- Gene Therapy - Volume 31 Issue 11-12, November 2024 - Nature.com - November 27th, 2024
- Iovance Biotherapeutics Announces the Promotion of Raj Puri, M.D., Ph.D. to Chief Regulatory Officer - GlobeNewswire - November 27th, 2024
- Patient Dies in Gene Therapy Trial, But FDA Permits Neurogene to Proceed With Low Dose - MedCity News - November 27th, 2024
- New CRISPR system pauses genes, rather than turning them off permanently - Livescience.com - November 27th, 2024
- Liver-targeting gene therapy lowers mice whole-body SMA symptoms - SMA News Today - November 27th, 2024
- Bright breakthroughs: Real stories of beating rare disease - Science - November 27th, 2024
- Sarepta Therapeutics Announces Global Licensing and Collaboration Agreement with Arrowhead Pharmaceuticals for Multiple Clinical and Preclinical siRNA... - November 27th, 2024