gene therapy

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FDA shares additional draft guidance on cell and gene therapy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
Astellas licenses AviadoBio’s dementia gene therapy candidate
Biopharma Reporter
Fri, 10/11/24 - 09:29 am
Astellas
AviadoBio
dementia
gene therapy
New data underline cancer risk of Bluebird therapy for brain disease
BioPharma Dive
Thu, 10/10/24 - 11:15 am
Bluebird Bio
gene therapy
Skysona
cALD
Astellas places $2bn+ bet on AviadoBio dementia gene therapy
Pharmaphorum
Tue, 10/8/24 - 09:44 am
Astellas
gene therapy
AviadoBio
frontotemporal dementia
Ultragenyx tweaks gene therapy dosing to dial up efficacy after seeing signs of activity
Fierce Biotech
Fri, 10/4/24 - 09:38 am
Ultragenyx Pharmaceutical
Wilson disease
gene therapy
UX701
Genespire raises €46.6 million (~$52 million) in a Series B round to advance its first pediatric in-vivo gene therapy into the clinic
Yahoo Finance
Wed, 09/25/24 - 11:58 am
Genespire
gene therapy
funding
pediatric
Bluebird bio lays off 25% of workforce as gene therapy pioneer struggles to stay afloat
Fierce Pharma
Tue, 09/24/24 - 11:35 am
Bluebird Bio
layoffs
gene therapy
4DMT’S Gene Therapy for AMD Clears Phase II, Heads to Late-Stage Studies
BioSpace
Thu, 09/19/24 - 11:07 am
4DMT
gene therapy
AMD
clinical trials
wet age-related macular degeneration

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