Articles

RARECast Podcasts
Rare Advocate
December 2, 2021
Rare Leader: Megan Golden, CEO and Co-Founder, Mission: Cure
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Finance
December 2, 2021
Krystal Biotech Raises $200 Million in Public Offering to Advance Redosable Gene Therapies
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Finance
December 2, 2021
AviadoBio Raises $80 million to Advance Neurodegenerative Gene Therapy Platform
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Deals
December 2, 2021
Astellas and Dyno Partner to Develop Next-Generation AAV Gene Therapy Vectors for Skeletal and Cardiac Muscle
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Finance
December 2, 2021
Tune Therapeutics Launches with Epigenomic Control Platform to Master Gene Networks
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Research
December 2, 2021
GBT Grants $400,000 to Help Expand Sickle Cell Real-World Data Gathering in United States
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Drug Development
December 1, 2021
Vertex Reports Positive Results from Phase 2 Study of VX-147 in APOL1-Mediated FSGS
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Drug Development
December 1, 2021
Neuvivo Launches to Develop and Market Effective and Safe Treatments for ALS
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Regulatory
November 30, 2021
FDA Issues Draft Guidance on Use of Registries to Support Regulatory Decision-Making
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News

August 12, 2021
Global Genes Grant Awardee Achieves Goal of Design...
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August 3, 2021
RARE Foundation Alliance Member Spotlight: Jennife...
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July 9, 2021
RARE Foundation Alliance Member Spotlight: Ashley ...
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June 24, 2021
RARE Foundation Alliance Member Spotlight: Matt La...
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May 10, 2021
RARE Foundation Alliance Member Spotlight: Sharon ...
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April 5, 2021
RARE Foundation Alliance Member Spotlight: Allison...
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