PEPGEN

In December 2024, the FDA approved Mesoblast’s remestemcel-L for steroid-refractory GvHD, placed a clinical hold on PepGen’s DMD trial, fast-tracked Sana Biotechnology’s SC291 for SLE, and agreed with uniQure on an accelerated approval pathway for Huntington disease gene therapy AMT-130.

In December 2024, the FDA made several key decisions: granted breakthrough therapy to STK-001 for Dravet syndrome, provided guidance for CNM-Au8 in ALS, aligned on AMT-130's accelerated approval for Huntington's, designated tolebrutinib for nrSPMS, placed a hold on PGN-EDO51 for DMD, and approved tirzepatide for OSA and obesity.

Novo Holdings completes $16.5B Catalent Pharma Solutions buyout. Merck pays $112M upfront for obesity pill. Ottimo Pharma raises $140M for cancer therapy. Novo Nordisk invests $1.2B in Denmark factory. Angitia Biopharmaceuticals raises $120M for musculoskeletal candidates. Novartis closes MorphoSys sites, laying off 330. SiteOne Therapeutics secures $100M for non-opioid pain drugs. Novo Nordisk's obesity drug underperforms. Bristol Myers Squibb invests $100M in BioArctic's Alzheimer antibodies. Sudan's healthcare collapses. Tonix Pharmaceuticals files NDA for fibromyalgia drug. Neurocrine Biosciences wins FDA approval for Crenessity. Edgewise Therapeutics discusses accelerated approval for Becker muscular dystrophy. FDA places partial hold on PepGen's Duchenne therapy. Checkpoint Therapeutics' immunotherapy approved. WHO opens Academy in Lyon. GSK's Jemperli earns FDA Breakthrough status. Sanofi's duvakitug shows remission in ulcerative colitis. Sandoz agrees to $275M settlement over drug pricing. Pfizer projects 2025 earnings. Mesoblast's Ryoncil approved as 1st MSC therapy for graft-vs-host disease.

FDA placed a clinical hold on PepGen's phase 2 CONNECT2-EDO51 study for Duchenne muscular dystrophy. Edgewise Therapeutics reported positive phase 2 CANYON trial results for sevasemten in Becker muscular dystrophy. Sanofi's tolebrutinib received breakthrough therapy designation for non-relapsing secondary progressive multiple sclerosis.

The FDA issued a clinical hold on PepGen's phase 2 trial for PGN-EDO51, delaying its US launch for Duchenne muscular dystrophy treatment. The hold does not affect ongoing studies outside the US. PepGen aims to address FDA questions expeditiously. PGN-EDO51, designed to skip exon 51, has Orphan Drug and Rare Pediatric Disease designations. Earlier study results showed positive exon skipping and dystrophin production.

The cell and gene therapy sectors are rapidly advancing, with updates on PepGen's PGN-EDO51 IND hold, innovative payment models for high-cost treatments, Janssen and Legend Biotech's Carvykti showing higher MRD negativity rates, Bristol Myers Squibb and 2seventy bio's Abecma responses in CNS-affected MM patients, Editas Medicine discontinuing EDIT-301 development, and SpliceBio's SB-007 receiving IND clearance for Stargardt disease treatment.

The FDA has placed a hold on PepGen's IND application for PGN-EDO51, a PMO for Duchenne muscular dystrophy, delaying the U.S. phase 2 CONNECT2-EDO51 trial. Meanwhile, the CONNECT1-EDO51 trial in Canada continues, with interim data showing positive results in exon skipping and dystrophin levels. PepGen aims to address FDA concerns to expedite CONNECT2-EDO51.

FDA placed a clinical hold on PepGen's phase 2 CONNECT2-EDO51 study for PGN-EDO51 in DMD patients. CONNECT1-EDO51 study continues in Canada, showing promising exon skipping and dystrophin production at 5 mg/kg dose with good tolerability.

Novo Nordisk to build new Denmark facility, Pepgen's Duchenne drug on FDA hold, Atea Pharmaceuticals hires Evercore, Neurocrine's Crenessity approved for CAH, Checkpoint's Unloxcyt for skin cancer, and Galderma's Nemluvio for eczema.

Global exosome market to grow at ~20% CAGR by 2030, driven by chronic disease incidence, diagnostic improvements, and personalized medicine interest. North America leads, with key players like Danaher and Thermo Fisher Scientific. Notable developments include Aruna Bio's US patent for neural exosomes and EXO Biologics' EUR 16 million Series A funding.