Scientists granted $420,000 to test gene therapy for rare neurological disease
Childhood-onset striatonigral degeneration is a rare genetic disorder that robs children of the ability to walk and speak by ...
Fierce Pharma2h
For struggling cell and gene therapy field, Peter Marks' FDA exit adds even more uncertainty
The sudden resignation of Peter Marks, M.D., Ph.D., from the FDA branch that regulates biological products for humans ...
Fierce Pharma7h
Gene therapy CDMO Artis BioSolutions launches with acquisition of Landmark Bio
Out of the shadows, new CDMO Artis BioSolutions has decloaked on a mission to tackle the tricky production process for ...
Fierce Biotech10h
Atsena raises $150M for ocular gene therapies with Bain, Sofinnova in tow
The $150 million will also go toward Atsena’s preclinical pipeline and expand the use of the biotech’s spreading AAV.SPR ...
Gene therapy loses luster as investors eye quicker returns from weight-loss drugs
Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing early investors to ...
11 Best Gene Therapy Stocks to Buy According to Analysts
Here is our list of the 11 Best Gene Therapy Stocks to Buy According to Analysts.
Clinical Trials Arena on MSN1d
Tenaya highlights HCM gene therapy interim data at ACC 2025
Three patients living with hypertrophic cardiomyopathy have had their disease classification downgraded following a single ...
News Medical on MSN19h
New study helps optimize gene therapy
Gene therapy, a technique that is revolutionizing the treatment of multiple genetic conditions, including eye and muscle diseases and blood disorders, requires efficient and specific delivery of the ...
BioPharma Dive2d
Cell, gene therapy makers lose a champion at FDA with exit of Peter Marks
Marks’ resignation leaves the field without a regulator many view as “integral” to its progress over the last decade.
Medera Presented Updated Results from First-In-Human Gene Therapy Trial for Heart Failure with Preserved Ejection Fraction at 2025 HFpEF Summit
Medera Inc. (“Medera”), a clinical-stage biopharmaceutical company focused on targeting cardiovascular diseases by developing ...
University of California13d
Breathing new life: Working toward an inhalable gene therapy for cystic fibrosis
Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...
Xcellbio Announces Commercial Licensing & Supply Agreement to Support Commercial Production of Gene Therapy for Sickle Cell disease
Xcellbio announced today that it had granted bluebird bio, Inc., a commercial license and entered into a supply agreement for the use of Xcellbio’s proprietary cell manufacturing technology, AVATAR™, ...