Articles

Finance
December 5, 2022
SonoThera Raises $61 Million To Advance Ultrasound-Guided, Nonviral Gene Therapy Platform
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Deals
December 5, 2022
MaxCyte Signs Strategic Platform License with Curamys to Enable Cell and Gene Therapies
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Drug Development
December 5, 2022
Ideaya Receives Fast Track Designation for Darovasertib with Crizotinib to Treat Rare Eye Melanoma
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Drug Development
December 5, 2022
FDA Lifts Clinical Hold on Beam’s Experimental Gene Editing to Treat Certain T-Cell Cancers
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Deals
December 5, 2022
J&J Says It Won’t Make a Bid for Horizon
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Drug Development
December 5, 2022
FDA Grants Breakthrough Therapy Designation for Syndax’s Revumenib to Treat Rare Leukemia
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RARECast Podcasts
December 2, 2022
Addressing the Current Limitations of AAV Gene Therapies
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Drug Development
December 2, 2022
FDA Rejects’ Y-mAbs’ Rare Pediatric Cancer Drug
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Finance
December 2, 2022
CIRM Grants $8.8 Million to Pursue Gene Therapies for Two Rare Diseases
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RARECast Podcasts
November 11, 2022
Helping Regulators and Drug Developers Understand the Challenges of Living with Fabry Disease
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News

October 28, 2022
Though she be but little, she is FIERCE
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October 26, 2022
RARE Global Advocacy Alliance Member Spotlight: Ch...
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October 12, 2022
Empowering the Next Generation Rare Disease Advoca...
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September 27, 2022
RARE Global Advocacy Alliance Member Spotlight: An...
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