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Aug 21, 2026
New research shows that many people with disease-causing gene mutations never develop illness, challenging traditional assumptions. Studies of 'modifier genes' and genetic outliers are revealing how some individuals stay healthy, potentially leading to new treatments for genetic diseases.
Apr 05, 2026
Biotech investors are closely watching cystic fibrosis drug trials, rare-disease therapies and regulatory catalysts. Sionna Therapeutics is advancing a new CF drug class, while Praxis Precision Medicines targets essential tremor. FDA staffing challenges add uncertainty to approval timelines.
Jul 18, 2026
4D Molecular Therapeutics (Nasdaq: FDMT) granted a total of 229,700 restricted stock units to 20 new non-executive hires across three awards in 2026 under its Employment Inducement Award Plan. The grants occurred on February 10, April 14, and July 14, as part of Nasdaq-compliant inducement awards.
May 04, 2026
Krystal Biotech said IOLITE enrollment for KB803 is complete and it remains on track for KB803 and KB801 registrational data readouts in 2026. The company also reported FDA platform technology designations for KB407 and KB111 and continued VYJUVEK expansion.
Apr 25, 2026
Researchers developed a lipid nanoparticle gene-editing approach that inserted a full CFTR gene into airway cells and restored 88% to 100% of normal channel function in a cystic fibrosis lab model.
Apr 18, 2026
A lipid nanoparticle gene-editing approach inserted a full healthy CFTR gene into human airway cells and restored 88% to 100% of normal CFTR function in lab tests. The nonviral strategy is intended as a mutation-agnostic path for cystic fibrosis.
Mar 20, 2026
Krystal Biotech posted Q4 2025 net revenue of $107.1 million from Vyjuvek sales, with full-year revenue reaching $389.1 million. The company expanded to over 90 patients across Germany, France, and Japan while advancing its pipeline with new FDA designations.
Mar 09, 2026
Three new CRISPR-based therapies show promise for treating heart failure through mitochondrial enhancement, cystic fibrosis via lipid nanoparticle gene insertion, and elevated cholesterol with single-dose gene editing targeting ANGPTL3.
Mar 02, 2026
UCLA scientists developed a lipid nanoparticle-based gene editing system that successfully inserts a full-length healthy CFTR gene into human airway cells, offering potential treatment for cystic fibrosis patients unresponsive to current therapies.
Feb 18, 2026
UCLA researchers developed lipid nanoparticle-based gene editing that inserts a complete CFTR gene into human airway cells, restoring up to 100% of normal channel function in cystic fibrosis laboratory models without viral vectors.
Dec 30, 2025
Drug Hunter's most-accessed resources, reviews, and case studies in 2025 reflected key trends in drug discovery, including the rise of oral macrocycles, advances in new modalities, and the shift from biologics to small molecules in immunology.
Feb 13, 2026
Vertex Pharmaceuticals reported fourth quarter 2025 revenue of $3.19 billion, up 10% year-over-year, and provided full year 2026 revenue guidance of $12.95 billion to $13.1 billion with over $500 million expected from non-CF products.
