Rare Diseases

Inovio, with path to market opening up, drafts accelerated approval talking points ahead of FDA meeting
Inovio, with path to market opening up, drafts accelerated approval talking points ahead of FDA meeting
Fierce Biotech
Inovio Pharmaceuticals
Rare Diseases
INO-3107
DNA
FDA
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FDA Launches Pilot Program to Help Further Accelerate Development of Rare Disease Therapies
FDA Launches Pilot Program to Help Further Accelerate Development of Rare Disease Therapies
Applied Clinical Trials Online
FDA
Rare Diseases
clinical trials
drug development
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DeepMind’s New AI Can Predict Genetic Diseases
DeepMind’s New AI Can Predict Genetic Diseases
Wired
Google
DeepMind
AlphaMissense
artificial intelligence
DNA
R&D
Rare Diseases
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Gene therapy consortium targets eight rare diseases for clinical trials
Gene therapy consortium targets eight rare diseases for clinical trials
BioPharma Dive
gene therapy
Rare Diseases
clinical trials
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CBER to Launch Operation Warp Speed for Rare Diseases by Year’s End
CBER to Launch Operation Warp Speed for Rare Diseases by Year’s End
BioSpace
CBER
Operation Warp Speed
FDA
Rare Diseases
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FDA plans to launch communications pilot for promising rare disease gene therapies
FDA plans to launch communications pilot for promising rare disease gene therapies
RAPS.org
FDA
biologics
gene therapy
Rare Diseases
regulatory
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Can the FDA keep the momentum going for rare disease drug approvals?
Can the FDA keep the momentum going for rare disease drug approvals?
Pharmaceutical Technology
FDA
Rare Diseases
drug approvals
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Pfizer pivots from early-stage rare disease R&D, shifting to external innovation and putting assets up for sale
Pfizer pivots from early-stage rare disease R&D, shifting to external innovation and putting assets up for sale
Fierce Biotech
Pfizer
R&D
externalization
gene therapy
Rare Diseases
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Acasti, seeking to expand use of old steroid, posts clinical data on oral spray in rare disease
Acasti, seeking to expand use of old steroid, posts clinical data on oral spray in rare disease
Fierce Pharma
Acasti Pharma
betamethasone
Rare Diseases
GTX-102
ataxia telangiectasia
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Rallybio, AbCellera partner to develop therapeutics for rare diseases
Rallybio, AbCellera partner to develop therapeutics for rare diseases
Pharmaceutical Business Review
Rallybio
AbCellera
Rare Diseases
drug discovery
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Drug Development for Rare Diseases is Littered with Regulatory Roadblocks
Drug Development for Rare Diseases is Littered with Regulatory Roadblocks
BioSpace
Rare Diseases
drug development
regulatory
KemPharm
Orphazyme
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Bouncing back from late-phase fail, Minoryx pockets €51M to guide rare disease drug to market
Bouncing back from late-phase fail, Minoryx pockets €51M to guide rare disease drug to market
Fierce Biotech
Minoryx
clinical trials
MIN-102
Rare Diseases
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FDA’s CDER Introduces New ARC Program to Accelerate Rare Disease Cures
FDA’s CDER Introduces New ARC Program to Accelerate Rare Disease Cures
Xtalks
FDA
CDER
ARC program
Rare Diseases
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Can FDA replicate Operation Warp Speed for rare diseases? Not yet, but Peter Marks has some ideas
Can FDA replicate Operation Warp Speed for rare diseases? Not yet, but Peter Marks has some ideas
Fierce Biotech
FDA
Peter Marks
CBER
gene therapy
cell therapy
Operation Warp Speed
Rare Diseases
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Takeda/Evozyne Partnership Headlines Hot Week for Gene Therapy Deals
Takeda/Evozyne Partnership Headlines Hot Week for Gene Therapy Deals
BioSpace
Takeda
Evozyne
gene therapy
Rare Diseases
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Digging into biopharma’s big buyout areas
Digging into biopharma’s big buyout areas
EP Vantage
M&A
oncology
CNS
Rare Diseases
cancer
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Backed by $100M, Star Therapeutics sets out to find constellations of rare disease
Backed by $100M, Star Therapeutics sets out to find constellations of rare disease
MedCity News
Star Therapeutics
Electra Therapeutics
autoimmune disease
Rare Diseases
sHLH
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Takeda taps a doctor turned patient—and spread-the-word artist—for rare disease awareness campaign
Takeda taps a doctor turned patient—and spread-the-word artist—for rare disease awareness campaign
Fierce Pharma
Takeda
pharma marketing
patients
Rare Diseases
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PDUFA VII is a step in the right direction for the rare disease community
PDUFA VII is a step in the right direction for the rare disease community
RAPS.org
FDA
regulatory
PDUFA VII
Rare Diseases
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Rare disease patients struggle for access to experimental drugs
Rare disease patients struggle for access to experimental drugs
Beckers Hospital Review
Rare Diseases
compassionate use guidelines
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