Rare Diseases

FDA scraps adcomm for Applied's rare disease drug ahead of November decision
Fierce Biotech
Wed, 09/18/24 - 01:56 pm
Applied Therapeutics
govorestat
FDA
Rare Diseases
FDA pushed to be more open, collaborative to accelerate rare disease R&D
Fierce Biotech
Fri, 09/13/24 - 09:43 am
FDA
innovation
Rare Diseases
Roche tops pharma rivals for reputation among rare disease patient groups
Medical Marketing and Media
Wed, 08/21/24 - 11:39 am
Roche
reputation
Rare Diseases
Biomarin
Amgen
FDA launches innovation hub to spur rare disease R&D
Pharmaphorum
Fri, 07/19/24 - 11:37 am
FDA
Rare Diseases
R&D
Moderna’s mRNA Candidate Joins FDA’s Accelerator Program for Rare Diseases
BioSpace
Thu, 06/6/24 - 11:39 am
Moderna Therapeutics
mRNA-3705
FDA
Rare Diseases
START
Sanofi pays $80M to add Roche-rivaling Fulcrum drug to phase 3 rare disease pipeline
Fierce Biotech
Mon, 05/13/24 - 11:25 am
Sanofi
Fulcrum Therapeutics
Roche
GSK
Rare Diseases
clinical trials
losmapimod
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

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