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Immunology

StockWatch: Horizon Soars on Talks with Amgen, J&J, Sanofi

Gene Therapy

Gene Therapy Briefs: Cure Rare Disease CEO’s Brother Died in Clinical Trial

prokaryotic polysomes

Rare and Neglected Diseases

Stoked About ASOs: Stoke Therapeutics Treats Rare CNS Diseases With RNA-Based Medicines

Rare and Neglected Diseases

Noncoding Variants Switch on “Disallowed” Regulatory Gene in Pancreas to Cause Rare Disease

Rare and Neglected Diseases

StockWatch: Astellas Hones Gene Therapy Focus with Taysha Stake

Genome Editing

Gene Therapy Pioneers Share Genome Editing Insights at Chardan Conference

GEN 40

StockWatch: After Bluebird’s Second Approval, Investors Are Looking for More

Rare and Neglected Diseases

Potential Gene Therapy Developed for Childhood Blinding Disease

Medicine doctor touching electronic medical record on tablet. DNA, capsules

Sponsored

The AI-Led Engine Advancing Our Understanding of Rare and Ultra-Rare Diseases

NY Mets owner at Citi Field

Neurological Disorders

StockWatch: Minerva’s a Home Run with N.Y. Mets Owner

DNA Test Sanger Sequencing

Rare and Neglected Diseases

Computational Approach Demonstrates Why Cystic Fibrosis Drugs Fall Short

Financing

StockWatch: Murder-for-Hire Indictment Wounds Enochian Shares; Company Cites ‘Promising Future’

C elegans, SEM

Rare and Neglected Diseases

Researchers Uncover Novel Insights into Rare Genetic Condition

Francis Collins

Rare and Neglected Diseases

Francis Collins Urges Gene Therapy Community to Scale Efforts to Tackle...

kriya therapeutics

Gene Therapy

Kriya Raises $270M Series C toward Fully Integrated Gene Therapy Engine

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