BioSpace

Longeveron’s shares crash as stem cell therapy flunks mid-stage cardio trial

With just over $10 million in cash available to fuel operations through the end of the year, Longeveron is undertaking a ...
BioSpaceOpinion

After Novartis’ devastating M&A losses, long live the executive willing to try

Big Pharma needs more—not fewer—bold bets on rare and difficult diseases after Novartis’ efforts to invest in muscular ...
BioSpace

Trialt Chief R&D Officer Dr. Yuri Kartashov Delivered Keynote at COG CRO Summit Europe

Presentation asked what clinical data management can safely delegate to AI — and what makes that delegation trustworthy ...
BioSpace

Webinar: Manufacturing in Turbulent Times

Learn what manufacturing challenges lie ahead, how to equip your team to protect your program from avoidable manufacturing ...
BioSpace

When vision fades: Rethinking wet AMD and the durability gap

In this episode of Denatured, you’ll hear Philip Jacob Rosenfeld, professor of Ophthalmology at the University of Miami ...
BioSpace

Ultragenyx Announces Approval of FAYUVI™ Gene Therapy, the First-Ever FDA-Approved Treatment for Sanfilippo Syndrome Type A (MPS IIIA)

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the U.S. Food and Drug Administration (FDA) granted standard full approval of FAYUVI™ (rebisufligene etisparvovec-hopf), also known ...
BioSpace

Phase 3 Pediatric EMPAVELI Data Published in Clinical Journal of the American Society of Nephrology Show Reduction in Proteinuria and Stabilized Kidney Function in Adolescents ...

EMPAVELI-treated adolescents achieved a 75% relative reduction in proteinuria within 6 months versus placebo ; EMPAVELI is the only approved treatment for adolescents with C3G and ...
BioSpace

Xenon Announces Azetukalner NDA Submission to FDA for Focal Seizures and Provides Update on Psychiatry Clinical Program

New Drug Application for azetukalner in focal seizures submitted to U.S. Food and Drug Administration; X-TOLE3 and X-ACKT continue to enroll focal seizure and PGTCS patients, respectively The Company ...
BioSpace

Sempresto Announces Positive FDA Type B Pre-IND Meeting Response for Smartphone-Integrated Epinephrine Auto-Injector

FDA Feedback Aligns with Sempresto’s Proposed Development Strategy and Clarifies Path Forward WESTON, Mass.--(BUSINESS WIRE)--Medical technology company Sempresto, developing a smartphone-integrated ...
BioSpace

argenx announces results of Extraordinary General Meeting of Shareholders and appointment of Thomas M. Brakel and Khurem Farooq as Non-Executive Directors

Amsterdam, the Netherlands – argenx SE (Euronext & Nasdaq: ARGX), a global immunology innovation company, today announced the results of the Extraordinary General Meeting of Shareholders and ...
BioSpace

Roche announces grand opening of its Innovation Center Boston to drive advancements in Cardiovascular, Renal and Metabolic diseases

Basel, 17 September 2026 - Roche (SIX: RO, ROP; OTCQX: RHHBY) announced today the grand opening of the Roche Genentech Innovation Center Boston, expanding Roche and Genentech’s US research presence in ...
BioSpace

Simulations Plus Expands FDA Adoption of ADMET Predictor® with New Licenses for the Cosmetics Regulatory Science Branch

(Nasdaq: SLP) (“Simulations Plus” or the “Company”), a global leader in model-informed and AI-accelerated drug development that advances biopharma innovation, today announced that the U.S. Food and ...