gene therapy

Roche reports positive topline outcomes from DMD treatment trial
Clinical Trials Arena
Tue, 01/28/25 - 10:24 am
Roche
Elevidys
DMD
gene therapy
clinical trials
5 questions facing emerging biotech in 2025
BioPharma Dive
Tue, 01/14/25 - 11:22 am
biotech
funding
China
IPOs
cell therapy
gene therapy
M&A
RegenXBio sells Hunter syndrome gene therapy's U.S., Asia commercial rights for $110M upfront
Fierce Biotech
Tue, 01/14/25 - 09:59 am
Regenxbio
Hunter Syndrome
gene therapy
Asia
Vertex pays Orna $65M to work on next-gen gene therapies for blood disorders
Fierce Biotech
Tue, 01/7/25 - 10:28 am
Vertex Pharmaceuticals
Orna Therapeutics
ReNAgade Therapeutics
gene therapy
sickle cell disease
transfusion-dependent beta thalassemia
Positive data could expand use of Novartis’ gene therapy for SMA
Clinical Trials Arena
Fri, 01/3/25 - 11:02 am
Novartis
gene therapy
SMA
Zolgensma
OAV101 IT
Novartis’ Phase III spinal muscular atrophy therapy trial meets primary endpoint
Clinical Trials Arena
Thu, 01/2/25 - 11:25 am
Novartis
clinical trials
gene therapy
spinal muscular atrophy
SMA
OAV101 IT
Sangamo shares plunge after Pfizer ends hemophilia A gene therapy deal
MSN/Reuters
Mon, 12/30/24 - 10:50 pm
Sangamo Therapeutics
Pfizer
hemophilia A
gene therapy
giroctocogene fitelparvovec
Sensorion encouraged by deafness gene therapy data
Pharmaphorum
Wed, 12/18/24 - 11:10 am
Sensorion
otoferlin-related hearing loss
hearing loss
gene therapy
clinical trials
SENS-501
Gene therapy uptake in sickle cell stays slow, despite patient interest
BioPharma Dive
Mon, 12/9/24 - 10:37 am
gene therapy
sickle cell disease
patients
Casgevy
Lyfgenia
Bluebird Bio
Vertex Pharmaceuticals
FDA considers 'regulatory action' for bluebird's Skysona after blood cancer reports
Fierce Pharma
Mon, 12/2/24 - 07:46 pm
Bluebird Bio
Skysona
gene therapy
FDA
blood cancer
cALD
Neurogene drops high-dose arm in Rett study after death
Pharmaphorum
Fri, 11/22/24 - 11:09 am
Neurogene
clinical trials
Rett Syndrome
patient death
gene therapy
NGN-401
Novartis buys Kate Therapeutics, eyeing $55B CGT market opportunity by 2030
BioSpace
Thu, 11/21/24 - 11:54 am
Novartis
Kate Therapeutics
M&A
gene therapy
FDA shares additional draft guidance on cell and gene therapy
Clinical Trials Arena
Wed, 11/20/24 - 11:20 am
FDA
regulatory
gene therapy
cell therapy
FDA endorses speedy approval path for Regenxbio Duchenne gene therapy
BioPharma Dive
Mon, 11/18/24 - 06:32 pm
Rengexbio
DMD
gene therapy
FDA
RGX-202
CSL Behring shuts down California cell and gene therapy R&D site amid group pivot
Fierce Biotech
Mon, 11/18/24 - 11:27 am
CSL Behring
California
gene therapy
cell therapy
R&D
Neurogene falls again on Rett gene therapy side effects
Pharmaphorum
Mon, 11/18/24 - 11:11 am
Neurogene
Rett Syndrome
clinical trials
gene therapy
Neurogene’s stock sinks after adverse event with Rett syndrome gene therapy
Clinical Trials Arena
Tue, 11/12/24 - 10:59 am
Neurogene
Rett Syndrome
gene therapy
clinical trials
Roche Inks Potential $1B Gene Therapy Deal With Dyno, Eyes Neurological Diseases
BioSpace
Thu, 10/24/24 - 08:21 pm
Roche
gene therapy
Dyno Therapeutics
neurological disorders
Pfizer lays off 75 more workers in North Carolina in wake of late-stage DMD trial fail
Fierce Pharma
Tue, 10/22/24 - 11:21 pm
Pfizer
layoffs
North Carolina
gene therapy
DMD
MeiraGTx plans to take Parkinson's gene therapy into phase 3
Pharmaphorum
Wed, 10/16/24 - 09:59 am
MeiraGTx
gene therapy
clinical trials
Parkinson's Disease
AAV-GAD

Pages