Ultragenyx Reports Promising Phase III Clinical Trial Results for Urea Cycle Disorder Treatment
Ultragenyx’s gene therapy DTX301, based on technology invented by Jim Wilson, MD, PhD, Emeritus Professor at Penn Medicine, helped control ammonia levels in patients with a urea cycle disorder.
Verismo Therapeutics Receives $28 million to Accelerate KIR-CAR Clinical Development
The funding from HLB Innovation, Verismo’s parent company, will advance clinical development of its killer cell immunoglobulin-like receptor (KIR)-chimeric antigen receptor (CAR) platform\.
Biogen to Acquire Apellis for $5.6B
Apellis’ C3 complement therapies, Syfovre® and Empaveli® will expand Biogen's presence in immunology.
Penn and CHOP Scientists Win 2026 Breakthrough Prize for Developing Gene Therapy for Inherited Blindness
These faculty developed the first FDA-approved gene therapy for an inherited condition.
Four Penn Medicine Studies Named Among Nation’s Top 10 Clinical Research Advances
The Clinical Research Forum’s Top 10 Clinical Research Achievement Awards honor outstanding accomplishments in clinical research.
Penn Medicine and CHOP Faculty Make the TIME100 Health List for CRISPR Gene Therapy Breakthrough
Two doctors were recognized by TIME as pioneers in healthcare for successfully treating KJ Muldoon.
Penn Received $7.8 Million from ARPA-H to Advance Lymphatic Imaging and AI-Driven Disease Detection
The project is led by Maxim Itkin, MD, Professor of Radiology at Penn Medicine and the director of Penn’s Center for Lymphatic Disorders.
Penn Expands its Immunology Research
Penn’s newly developed seven-story facility at 3600 Civic Center Boulevard will bring together labs across disciplines to advance breakthroughs in immune health, autoimmunity, and infectious diseases.
Dispatch Bio Developing Tumor-Agnostic Immunotherapy Targeting Solid Tumors
The technology, now in preclinical development, has the potential to overcome the limitations of traditional cancer treatments for solid tumors.
Opus Genetics Reports Positive Pediatric Data from its Clinical Trial Evaluating Investigational Gene Therapy for a Rare Eye Disorder
These findings suggest OPGx-LCA5, Opus Genetics' investigational gene augmentation therapy, could improve vision for patients previously considered untreatable.