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 ...
Gene therapy reverses symptoms of rare neurological disease in animal models
A recent study led by the UAB demonstrates the potential of gene therapy to restore motor capacity in an ultra-rare disease, ...
BioPharma Dive13h
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.
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.
Pharmaceutical Technology20h
Cell and gene therapy collections: The need for consistency
For cell and gene therapy, a consistent supply of quality starting material is vital that is maintained throughout the ...
Fierce Pharma20h
As FDA's Peter Marks resigns, industry watchers size up vaccine, cell and gene therapy hits
As the FDA wavers from cuts, changes and departures under the second Trump administration, another vital figure of the agency ...
Teen’s death following Sarepta DMD gene therapy underscores a risk seen for decades
The death of a 16-year-old boy evokes a lengthy battle to realize the benefit of cutting-edge gene therapy while also ...
University of California11d
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 ...
Gene-Therapy Maker Sarepta Tanks After Patient Death
Shares of Sarepta Therapeutics have struggled for the past year due to concerns about the commercial potential of its gene ...
The American Journal of Managed Care13d
Bustling Gene Therapy Pipeline for Neuromuscular Diseases Brings Thorny Questions to the Clinic
The rapid development of gene therapy options for treating neuromuscular diseases has created new therapeutic options but ...
AlphaGalileo4h
Unlocking therapeutic potential: antibacterial therapy for CRB1-associated retinal degeneration in Rd8 mice
Inherited retinal degeneration (IRD) encompasses various disorders characterized by progressive loss of retinal photoreceptor cells, ultimately ...