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478,848 Results for "jaguar gene therapy".
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Gene therapy
5 Cardio Gene Therapies on the Near Horizon
Benefiting from technological and conceptual groundwork and positive early data, gene therapies are advancing in the clinic for cardiovascular diseases including congestive heart failure, chronic refractory angina and cardiomyopathy.
January 13, 2025
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9 min read
·
Tristan Manalac
Press Releases
Significant Positive Results with Jaguar Health’s Crofelemer for Cancer Therapy-Related Diarrhea (CTD) in Breast Cancer Patients Presented at San Antonio Breast Cancer Symposium (SABCS)
December 12, 2024
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8 min read
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
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2 min read
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Kate Goodwin
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
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7 min read
Neurodegenerative disease
UniQure Clears Path to Accelerated Approval for Huntington’s Gene Therapy
In a Type B meeting, the FDA signified that it will allow uniQure to use a natural history control, the composite Unified Huntington’s Disease Rating Scale, and neurofilament light chain levels to support the accelerated approval of its gene therapy AMT-130.
December 10, 2024
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2 min read
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Tristan Manalac
Career Advice
The Right Fit: What BMS, Intellia Look for in Cell and Gene Therapy Talent
When hiring job candidates to work on cell and gene therapies, companies look for more than just technical skills. Talent acquisition executives from Bristol Myers Squibb and Intellia Therapeutics offer an inside look at what they want in an employee.
December 12, 2024
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5 min read
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Angela Gabriel
Drug pricing
Bluebird, Vertex/CRISPR’s SCD Gene Therapies Get New CMS Payment Model
The payment scheme will tie gene therapy payments to improvements in health outcomes—and could potentially boost the uptake of these sickle cell disease treatments.
December 5, 2024
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2 min read
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Tristan Manalac
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
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2 min read
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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
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2 min read
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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
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2 min read
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Tristan Manalac
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