gene therapy

ASH: Despite partial clinical hold, bluebird's sickle cell gene therapy remains on track
Fierce Pharma
Sun, 12/11/22 - 01:34 pm
Bluebird Bio
ASH 2022
Zynteglo
sickle cell disease
gene therapy
lovo-cel
Despite Eye-Popping $3.5 Million Price Tag For Gene Therapy Hemgenix, Budget Impact For Most Payers Will Be Relatively Small
Forbes
Mon, 12/5/22 - 10:48 am
Hemgenix
gene therapy
hemophilia B
drug pricing
CSL
Do Verve's gene edits pass down to patients' children? The FDA wants to know
Fierce Biotech
Mon, 12/5/22 - 10:42 am
Verve Therapeutics
gene editing
gene therapy
FDA
VERVE-101
CTAD 2022 – Lexeo takes baby steps towards an Alzheimer’s gene therapy
EP Vantage
Sun, 12/4/22 - 01:07 pm
Lexeo
Alzheimer's disease
CTAD
gene therapy
Bristol Myers has change of heart on cardiovascular gene therapy pact, terminating uniQure deal
Fierce Biotech
Tue, 11/29/22 - 10:57 am
Bristol Myers Squibb
uniQure
gene therapy
uniQure, CSL claim first okay for hemophilia B gene therapy
Pharmaphorum
Wed, 11/23/22 - 07:53 am
uniQure
CSL
hemophila B
gene therapy
Hemgenix
FDA
Lysogene's lead gene therapy flunks phase 2/3 trial almost a year after Sarepta walked out
Fierce Biotech
Sun, 11/20/22 - 12:54 pm
Lysogene
gene therapy
clinical trials
LYS-SAF302
Sanfillipo Syndrome
Novartis cuts 275 jobs at Illinois site to consolidate Zolgensma manufacturing in North Carolina
Endpoints
Wed, 11/9/22 - 11:13 pm
Novartis
layoffs
gene therapy
Zolgensma
drug manufacturing
ASH preview – waiting for Editas
EP Vantage
Wed, 11/9/22 - 10:40 am
ASH 2022
sickle cell disease
Bluebird Bio
Sangamo BioSciences
Novartis
Editas Medicine
gene therapy
Vertex Pharmaceuticals
Sanofi
BioMarin's hemophilia gene therapy faces further delays over fresh FDA requests
Fierce Biotech
Tue, 11/8/22 - 10:46 am
Biomarin
gene therapy
FDA
hemophilia
Roctavian
FDA finalizes umbrella trial guidance for cell and gene therapies
RAPS.org
Sun, 11/6/22 - 01:28 pm
FDA
regulatory
clinical trials
umbrella trials
gene therapy
cell therapy
Sarepta preps $1.2B war chest as DMD gene therapy nears accelerated FDA nod
Fierce Biotech
Thu, 11/3/22 - 11:56 am
Sarepta Therapeutics
Duchenne Muscular Dystrophy
SRP-9001
gene therapy
Cost watchdog ICER backs multimillion-dollar price tags for BioMarin, CSL hemophilia gene therapies
Fierce Pharma
Thu, 11/3/22 - 11:02 am
ICER
drug pricing
gene therapy
Biomarin
CSL
Bluebird Bio
hemophilia
Editas Awaits Data to Determine the Future of Ophthalmic Gene Therapy, EDIT-101
BioSpace
Wed, 11/2/22 - 10:54 pm
Editas Medicine
EDIT-101
gene therapy
Leber's congenital amaurosis
UniQure revs up engines again for Huntington's gene therapy after trial hospitalizations
Fierce Biotech
Wed, 11/2/22 - 10:49 pm
uniQure
Huntington's disease
gene therapy
AMT-130
String of gene therapy deals spurs cautious optimism on Wall Street
BioPharma Dive
Tue, 11/1/22 - 07:09 pm
gene therapy
M&A
Akouos
AGTC
Taysha Gene Therapies
Astellas
Eli Lilly
Syncona
Replay Launches Eudora to Target Genetic Retinal Diseases
BioSpace
Tue, 11/1/22 - 12:04 pm
Replay
Eudora
gene therapy
retinal diseases
An unusual alliance opens a gene and cell therapy hub outside Boston
BioPharma Dive
Thu, 10/27/22 - 08:29 pm
Boston
cell therapy
gene therapy
Landmark Bio
Astellas Pays $50M for Access to Taysha's Rett, GAN Programs
BioSpace
Wed, 10/26/22 - 10:29 am
Taysha Gene Therapies
Astellas
gene therapy
Rett Syndrome
GAN
Novartis sales of Zolgensma gene therapy slow as market shifts
BioPharma Dive
Tue, 10/25/22 - 11:02 pm
Novartis
gene therapy
spinal muscular atrophy
SMA
Zolgensma

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