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Rare diseases
Acadia Scraps Prader-Willi Drug After Late-Stage Failure
Acadia Pharmaceuticals was testing the drug, an intranasal formulation of the oxytocin analogue carbetocin, for its potential to ease hyperphagia in the rare neurological condition.
September 24, 2025
·
1 min read
·
Dan Samorodnitsky
Venture capital
Sanofi Fattens Venture Unit With $625M Infusion for Rare Disease, Neuro Innovation
Sanofi Ventures, which now has $1.4 billion in total assets, will focus its investment efforts on early players working in immunology, rare diseases, neurology and vaccines.
September 24, 2025
·
2 min read
·
Tristan Manalac
Huntington’s disease
UniQure ‘Exceeds Expectations’ in Much-Anticipated 3-Year Huntington’s Readout
If approved, uniQure’s gene therapy AMT-130—which slowed disease progression by 75%—would be the first genetic treatment for Huntington’s disease. A BLA submission is planned for the first quarter of 2026.
September 24, 2025
·
2 min read
·
Heather McKenzie
Press Releases
Therma Bright Expands Relationship with Distribution Partner Gen-X Med for Venowave(TM) Sales
Initial 200-Unit Sale previously announced on April 24, 2025, Targeted Specialized Knee Surgeons in U.S. Healthcare Network
September 24, 2025
·
4 min read
Press Releases
Envoy Medical Advances Commercialization Planning for Breakthrough Hearing Device as FDA Clinical Trial Remains On Track
The Company Begins Strategic Preparations To Support Anticipated Demand And Optimization Of The Acclaim® Fully Implanted Cochlear Implant Opportunity Six-month follow-up visits are now underway and will continue over the next two months
September 24, 2025
·
8 min read
Press Releases
Kneat Recognised as Leader in G2 Pharma and Biotech Fall 2025 Grid Reports
Score based on User Satisfaction and Market Presence significantly exceeds peers
September 24, 2025
·
2 min read
Press Releases
Independent Research Demonstrates Ruvidar(R) Effective in the Destruction of Herpes Simplex Virus
September 24, 2025
·
9 min read
Manufacturing
FDA Hits J&J With Warning Letter Over ‘Significant Violations’ at Korean Production Plant
The issues the regulator found include the failure to comprehensively review complaints and product defects.
September 24, 2025
·
2 min read
·
Tristan Manalac
Manufacturing
Lilly Unveils Second Facility in Manufacturing Push, Naming Texas as Site of New $6.5B Plant
Lilly expects to open 615 high-wage roles in Texas in connection with its new facility, plus around 4,000 construction roles.
September 24, 2025
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2 min read
·
Tristan Manalac
Spinal muscular atrophy
Biogen Receives Surprise FDA Rejection for High Dose Spinraza
The regulatory action marks the second rejection for a spinal muscular atrophy therapy this week after Scholar Rock’s apitegromab was issued a complete response letter on Tuesday, similarly on manufacturing grounds.
September 24, 2025
·
2 min read
·
Tristan Manalac
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