gene therapy

Bluebird Boasts Nearly 140% Revenue Jump, Still Misses Target
BioSpace
Fri, 05/10/24 - 11:10 am
Bluebird Bio
earnings
gene therapy
In gene therapy race, bluebird leads Vertex in activating treatment centers but trails in patient starts
Fierce Pharma
Thu, 05/9/24 - 07:17 pm
gene therapy
Vertex Pharmaceuticals
Bluebird Bio
ASGCT24: Regeneron’s Gene Therapy Shows ‘Dramatic’ Hearing Improvement in Deaf Kids
BioSpace
Wed, 05/8/24 - 11:33 am
Regeneron
DB-OTO
genetic deafness
gene therapy
ASGCT
Sarepta Seeks Label Expansion, Bigger Market for DMD Gene Therapy
BioSpace
Wed, 05/8/24 - 11:13 am
Sarepta Therapeutics
DMD
Elevidys
gene therapy
Krystal Biotech touts topical gene therapy uptake, plots overseas launches
Fierce Pharma
Tue, 05/7/24 - 06:27 pm
Krystal Biotech
Vyjuvek
dystrophic epidermolysis bullosa
gene therapy
Verve moves forward with backup base editing therapy
BioPharma Dive
Tue, 05/7/24 - 11:29 am
Verve Therapeutics
clinical trials
gene therapy
heart disease
Vertex files FDA approval ask for next-gen cystic fibrosis candidate, resumes diabetes cell therapy trial
Fierce Biotech
Tue, 05/7/24 - 11:14 am
Vertex Pharmaceuticals
cystic fibrosis
Vanza Triple
FDA
gene therapy
First Patient Begins Newly Approved Sickle Cell Gene Therapy
NY Times, NY
Mon, 05/6/24 - 11:40 am
Bluebird Bio
gene therapy
sickle cell disease
patients
Lyfgenia
5 DMD Data Readouts to Watch in 2024
BioSpace
Mon, 05/6/24 - 11:24 am
DMD
clinical trials
Pfizer
gene therapy
fordadistrogene movaparvovec
Dyne Therapeutics
DYNE-251
Edgewise Therapeutics
EDG-5506
Capricor
CAP-1002
Avidity Biosciences
AOC 1044
FDA’s Accelerated Approval of Sarepta DMD Gene Therapy Comes Under Fire
BioSpace
Fri, 05/3/24 - 11:20 am
FDA
accelerated approvals
Sarepta Therapeutics
DMD
gene therapy
Pfizer hemophilia gene therapy arrives in US to uncertain future
BioPharma Dive
Fri, 04/26/24 - 11:15 am
Pfizer
gene therapy
FDA
hemophila B
Beqvez
Walgreens rolls out gene, cell therapy services
Beckers Hospital Review
Thu, 04/25/24 - 05:51 pm
Walgreens
cell therapy
gene therapy
Walgreens Specialty Pharmacy
AbbVie and Regenxbio’s ‘multibillion-dollar opportunity’ in vision loss gene therapy
Pharma Voice
Wed, 04/24/24 - 08:42 am
Regenxbio
AbbVie
gene therapy
ABBV-RGX-314
wet age-related macular degeneration
diabetic retinopathy
5 Cell and Gene Therapy Decisions to Watch in 2024
BioSpace
Mon, 04/22/24 - 11:38 am
cell therapy
gene therapy
Pfizer
Beqvez
hemophilia B
Abeona Therapeutics
pz-cel
recessive dystrophic epidermolysis bullosa
Rocket Pharmaceuticals
Kresladi
Leukocyte adhesion deficiency-1
Adaptimmune
afami-cel
advanced synovial sarcoma
Autolus Therapeutics
obe-cel
B cell acute lymphoblastic leukemia
FDA fast-tracks AskBio’s heart failure gene therapy
Pharmaphorum
Thu, 04/18/24 - 11:14 am
FDA
Bayer
AskBio
heart failure
gene therapy
fast track
AB-1002
Traditional gene therapies are uber-niche. Ocugen hopes to change that.
Pharma Voice
Wed, 04/17/24 - 11:14 am
gene therapy
Ocugen
Luxturna
Nanoscope eyes market after gene therapy restores vision for patients with retinal disease
Fierce Biotech
Tue, 03/26/24 - 10:53 am
Nanoscope Therapeutics
gene therapy
FDA
Orchard sets new gene therapy price tag at $4.25M—the steepest of any drug in the US
Fierce Pharma
Wed, 03/20/24 - 11:16 am
Orchard Therapeutics
drug pricing
metachromatic leukodystrophy
gene therapy
Bluebird bio inks first Medicaid coverage agreement for sickle cell gene therapy, signing on with Michigan
Fierce Pharma
Mon, 03/11/24 - 11:33 am
Bluebird Bio
Michigan
lovo-cel
Lyfgenia
Medicaid
sickle cell disease
gene therapy
Luigi Naldini: Gene therapy no longer a ‘dream’ but big pharma must step up
Biopharma Reporter
Thu, 03/7/24 - 05:22 pm
gene therapy
Big Pharma

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