Gene editing takes centre stage in FDA’s new rare disease approval pathway

"Gene editing takes centre stage in FDA’s new rare disease approval pathway" was originally created and published by ...

Children's Hospital of Philadelphia Marks One-Year Anniversary of World's First Personalized CRISPR Gene Therapy for Child with Rare Genetic Disease

February 25, 2026 marks one year since KJ, an infant born with severe carbamoyl phosphate synthetase 1 (CPS1) deficiency, became the world's first person to receive a personalized CRISPR-based gene ...

Scientists create world’s first tomatoes that smell like ‘buttered popcorn’

Scientists generate world’s first ’extraordinary aromatic tomato plants’ by simultaneously altering two key genes ...

Beam Therapeutics Reports Fourth Quarter and Year-End 2025 Financial Results and Announces New Liver-Targeted Genetic Disease Program in Phenylketonuria (PKU)

New Program Designed as Platform-based Approach for Direct Correction of Mutations Causing PKU; Investigational New Drug (IND ...
WVUA 23

FDA proposes new system for approving customized drugs and therapies for rare diseases

The Food and Drug Administration is proposing a system for approving customized drugs and medical treatments for patients ...

Intellia Therapeutics to Participate in Upcoming Investor Conferences

Intellia Therapeutics, Inc. (Nasdaq: NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine ...
PharmTech

Impacts of FDA's Plausible Mechanism Framework for Ultra-Rare Disease Therapies

A structured pathway supports individualized RNA-based and genome-editing therapeutics when ultra-rare prevalence precludes ...

Scientists make tomatoes that smell like ‘buttered popcorn’

Chinese scientists have developed a new tomato variety with a distinctive "popcorn-like" aroma using advanced gene editing ...

Beam Therapeutics Announces $500 Million Strategic Financing Facility with Sixth Street

Million Funded at Close with up to an Additional $400 Million Available Under Facility with Seven-Year TermFinancing Bolsters Balance Sheet ...

Therna Biosciences Appoints Biotech Veteran Devyn Smith, Ph.D., to Board of Directors and Expands Strategic and Scientific Advisory Leadership

"Devyn brings a rare combination of scientific depth, operational leadership, and strategic perspective," said Nazli Azimi, Ph.D., Chief Executive Officer of Therna. "His experience across genetic ...