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» Rare Diseases
Rare Diseases
Rare Disease Day Reminds Biopharma of Opportunities, Challenges
BioSpace
Fri, 03/1/24 - 11:43 am
Rare Diseases
Leveraging tRNA to Scale Genetic Medicines for Rare Diseases and Beyond
BioSpace
Mon, 12/11/23 - 09:56 am
tRNA
drug development
Rare Diseases
Inovio, with path to market opening up, drafts accelerated approval talking points ahead of FDA meeting
Fierce Biotech
Fri, 11/10/23 - 10:08 am
Inovio Pharmaceuticals
Rare Diseases
INO-3107
DNA
FDA
FDA Launches Pilot Program to Help Further Accelerate Development of Rare Disease Therapies
Applied Clinical Trials Online
Sun, 10/1/23 - 02:30 pm
FDA
Rare Diseases
clinical trials
drug development
DeepMind’s New AI Can Predict Genetic Diseases
Wired
Tue, 09/19/23 - 10:59 am
Google
DeepMind
AlphaMissense
artificial intelligence
DNA
R&D
Rare Diseases
Gene therapy consortium targets eight rare diseases for clinical trials
BioPharma Dive
Tue, 05/16/23 - 10:57 pm
gene therapy
Rare Diseases
clinical trials
CBER to Launch Operation Warp Speed for Rare Diseases by Year’s End
BioSpace
Mon, 04/24/23 - 11:36 am
CBER
Operation Warp Speed
FDA
Rare Diseases
FDA plans to launch communications pilot for promising rare disease gene therapies
RAPS.org
Mon, 02/27/23 - 07:10 pm
FDA
biologics
gene therapy
Rare Diseases
regulatory
Can the FDA keep the momentum going for rare disease drug approvals?
Pharmaceutical Technology
Mon, 01/23/23 - 09:57 pm
FDA
Rare Diseases
drug approvals
Pfizer pivots from early-stage rare disease R&D, shifting to external innovation and putting assets up for sale
Fierce Biotech
Fri, 01/6/23 - 10:09 am
Pfizer
R&D
externalization
gene therapy
Rare Diseases
Acasti, seeking to expand use of old steroid, posts clinical data on oral spray in rare disease
Fierce Pharma
Tue, 01/3/23 - 10:40 am
Acasti Pharma
betamethasone
Rare Diseases
GTX-102
ataxia telangiectasia
Rallybio, AbCellera partner to develop therapeutics for rare diseases
Pharmaceutical Business Review
Fri, 12/2/22 - 10:54 am
Rallybio
AbCellera
Rare Diseases
drug discovery
Drug Development for Rare Diseases is Littered with Regulatory Roadblocks
BioSpace
Fri, 08/19/22 - 10:41 am
Rare Diseases
drug development
regulatory
KemPharm
Orphazyme
Bouncing back from late-phase fail, Minoryx pockets €51M to guide rare disease drug to market
Fierce Biotech
Tue, 05/31/22 - 10:44 am
Minoryx
clinical trials
MIN-102
Rare Diseases
FDA’s CDER Introduces New ARC Program to Accelerate Rare Disease Cures
Xtalks
Wed, 05/11/22 - 10:41 pm
FDA
CDER
ARC program
Rare Diseases
Can FDA replicate Operation Warp Speed for rare diseases? Not yet, but Peter Marks has some ideas
Fierce Biotech
Wed, 05/11/22 - 10:29 am
FDA
Peter Marks
CBER
gene therapy
cell therapy
Operation Warp Speed
Rare Diseases
Takeda/Evozyne Partnership Headlines Hot Week for Gene Therapy Deals
BioSpace
Wed, 04/6/22 - 07:37 pm
Takeda
Evozyne
gene therapy
Rare Diseases
Digging into biopharma’s big buyout areas
EP Vantage
Fri, 03/11/22 - 10:26 am
M&A
oncology
CNS
Rare Diseases
cancer
Backed by $100M, Star Therapeutics sets out to find constellations of rare disease
MedCity News
Wed, 02/16/22 - 10:43 am
Star Therapeutics
Electra Therapeutics
autoimmune disease
Rare Diseases
sHLH
Takeda taps a doctor turned patient—and spread-the-word artist—for rare disease awareness campaign
Fierce Pharma
Sat, 02/5/22 - 12:02 pm
Takeda
pharma marketing
patients
Rare Diseases
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