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475,102 Results for "jaguar gene therapy".
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Pipeline
Neurogene Drops Batten Gene Therapy Program
After failing to receive the RMAT designation from the FDA for its early-stage Batten disease gene therapy, Neurogene tells investors that it’s evaluating options for the program.
November 19, 2024
·
2 min read
·
Kate Goodwin
Approvals
PTC Wins First FDA Nod for Direct-to-Brain Gene Therapy, Targets Ultrarare Disease
The FDA has followed in the footsteps of its European counterparts and granted accelerated approval to PTC Therapeutics’ gene therapy Kebilidi for AADC deficiency. It is the first approved gene therapy to be delivered directly to the brain.
November 14, 2024
·
2 min read
·
Tristan Manalac
Gene therapy
Neurogene Details Gene Therapy Complication, Sending Shares Plummeting Again
Neurogene’s shares fell by 36% as the market opened Monday morning following news that a patient experienced systemic hyperinflammatory syndrome in a Phase I/II clinical trial of Rett syndrome gene therapy NGN-401.
November 18, 2024
·
2 min read
·
Annalee Armstrong
Duchenne muscular dystrophy
Regenxbio Gears Up to Challenge Sarepta in DMD as Gene Therapy Advances to Pivotal Studies
Regenxbio is pushing its Duchenne muscular dystrophy gene therapy into pivotal development, with a BLA planned for 2026—potentially posing a threat to Sarepta’s Elevidys.
November 19, 2024
·
2 min read
·
Tristan Manalac
BioMidwest
Jaguar Gene Therapy Announces FDA Clearance of IND to Study JAG201 in a Genetic Form of Autism Spectrum Disorder and Phelan-McDermid Syndrome
Jaguar Gene Therapy today announced the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) Application for JAG201, a gene therapy for a genetic form of autism spectrum disorder (ASD) and Phelan-McDermid syndrome (PMS).
January 31, 2024
·
7 min read
Press Releases
Jaguar Gene Therapy to Initiate Inaugural Pediatric Clinical Trial Targeting a Genetic Form of Autism Spectrum Disorder and Phelan-McDermid Syndrome
July 9, 2024
·
6 min read
Editorial
Eyes, Ears Get Their Due With Cell and Gene Therapy
With promising early results, cell and gene therapies are making headway against both rare and common ocular and auditory diseases.
September 3, 2024
·
4 min read
·
Heather McKenzie
Gene therapy
Roche Inks Potential $1B Gene Therapy Deal With Dyno, Eyes Neurological Diseases
With an upfront payment of $50 million from Roche, the partnership will leverage Dyno Therapeutics’
in vivo
gene therapy delivery technology, which synthesizes virus capsids with better functionality and manufacturability.
October 24, 2024
·
2 min read
·
Tristan Manalac
Press Releases
Jaguar Health Reports Third Quarter 2024 Financial Results
November 13, 2024
·
13 min read
Layoffs
Astellas Gene Therapies to Close Biomanufacturing Facility, Affecting About 100 Employees
Astellas Gene Therapies is closing its San Francisco biomanufacturing facility, shifting gene therapy manufacturing to North Carolina, cutting at least 17 employees and affecting dozens more.
September 3, 2024
·
1 min read
·
Angela Gabriel
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