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» sickle cell disease
sickle cell disease
Forma Therapeutics gets European orphan drug status for FT-4202 to treat sickle cell disease
Pharmaceutical Business Review
Thu, 11/12/20 - 10:40 am
Forma Therapeutics
orphan drugs
Europe
FT-4202
sickle cell disease
Can bluebird bio Recover From Another Major Setback?
Motley Fool
Fri, 11/6/20 - 10:00 am
Bluebird Bio
bb1111
sickle cell disease
FDA scuttles bluebird's accelerated plans for sickle cell gene therapy, as higher CMC bar forces 1-year delay
Endpoints
Thu, 11/5/20 - 11:05 am
Bluebird Bio
FDA
sickle cell disease
gene therapy
HGB-206
CRISPR and Vertex Release Additional Data for Blood Disease Treatment
Motley Fool
Wed, 11/4/20 - 10:50 pm
CRISPR Therapeutics
CRISPR
Vertex Pharmaceuticals
CTX001
beta thalassemia
sickle cell disease
Forma takes aim at Agios with new AML complete remission data
Endpoints
Mon, 10/26/20 - 11:01 am
Forma Therapeutics
sickle cell disease
clinical trials
Agios
AML
Cyclerion fails sickle cell test, prompting pivot to CNS disease
Fierce Biotech
Wed, 10/14/20 - 10:46 am
Cyclerion
olinciguat
sickle cell disease
CNS
IW-6463
Forma Therapeutics nabs new R&D execs as it plots late-stage sickle cell test next year
Fierce Biotech
Tue, 09/15/20 - 11:32 pm
Forma Therapeutics
R&D
sickle cell disease
This Startup Might Finally Cure Sickle Cell Disease—After A Century Of Racist Neglect
Forbes
Fri, 07/10/20 - 10:54 am
Beam Therapeutics
sickle cell disease
racism
CRISPR and Vertex show durability of gene-editing therapy, hoping for one and done treatment
Fierce Biotech
Mon, 06/15/20 - 10:35 am
Vertex Pharmaceuticals
CRISPR Therapeutics
CTX001
gene editing
sickle cell disease
beta thalassemia
Magenta, Beam partner to explore use of antibody-based conditioning drug for gene therapies
MedCity News
Mon, 06/15/20 - 10:25 am
Magenta Therapeutics
Beam Therapeutics
MGTA-117
gene therapy
sickle cell disease
beta thalassemia
New CRISPR, gene therapy results strengthen potential for treatment of blood diseases
BioPharma Dive
Fri, 06/12/20 - 11:11 am
CRISPR
gene therapy
beta thalassemia
sickle cell disease
CRISPR Therapeutics
Vertex
Bluebird Bio
EHA
EHA 2020 – Bluebird’s sickle cell plan takes shape, with a stark reminder
EP Vantage
Fri, 06/12/20 - 10:41 am
Bluebird Bio
sickle cell disease
LentiGlobin
EHA
New sickle cell drugs priced too high, ICER says
BioPharma Dive
Fri, 03/13/20 - 12:43 pm
sickle cell disease
drug pricing
ICER
Novartis
Global Blood Therapeutics
RWE
Adakveo
Oxbryta
Blood disorder startup files for $86M IPO
Bizjournals
Wed, 02/19/20 - 09:52 am
IPOs
Imara
sickle cell disease
beta thalassemia
Sickle Cell Disease: After Years Of Neglect Some Promise For Sufferers
Forbes
Mon, 02/17/20 - 10:26 am
sickle cell disease
Oxbryta
Adakveo
Endari
olinciguat
CRISPR
GBT takes a page from HIV playbook as it embarks on Oxbryta launch
Fierce Pharma
Thu, 02/13/20 - 11:31 pm
Global Blood Therapeutics
drug launches
Oxbryta
sickle cell disease
HIV
Pfizer uncouples from GlycoMimetics and its failed sickle cell disease drug
Endpoints
Tue, 02/11/20 - 10:39 am
Pfizer
GlycoMimetics
sickle cell disease
Pfizer cans early-stage trials for cancer, sickle cell targets
Fierce Biotech
Tue, 01/28/20 - 11:56 am
Pfizer
sickle cell disease
cancer
Trifecta of sickle cell disease therapies extend life expectancy, but are not cost-effective — ICER
Endpoints
Fri, 01/24/20 - 10:30 am
ICER
sickle cell disease
Novartis
Adakveo
Global Blood Therapeutics
Oxbryta
Emmaus Life Sciences
Endari
3 Rare Disease Drug Launches to Watch in 2020
Motley Fool
Sat, 01/4/20 - 07:38 pm
Global Blood Therapeutics
sickle cell disease
Oxbryta
Novartis
SCD
Adakveo
Alnylam
Givlaari
acute hepatic porphyria
FDA
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