PepGen’s Low-Dose DM1 Data Falls Flat: Minimal Splicing Correction and No Clear Functional Benefit
PepGen’s latest low-dose (5 mg/kg) DM1 data shows minimal splicing correction and almost no functional improvement over placebo.
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PepGen Faces FDA Partial Hold on FREEDOM2 DM1 Trial, Advances in Multiple Countries
PepGen announces FDA partial clinical hold on FREEDOM2-DM1 trial due to preclinical questions, while gaining clearance in South Korea, Australia, New Zealand.
Novartis Makes Bold $12 Billion Move with Avidity Biosciences Acquisition in Largest Deal Under CEO Narasimhan
Novartis acquires Avidity Biosciences for $12 billion, gaining access to breakthrough RNA therapeutics for neuromuscular diseases as the Swiss drugmaker prepares for upcoming patent expirations on key drugs.
PepGen Shares Double after Muscle Disease Drug Shows Promise in Trial
PepGen's DM1 drug (PGN-EDODM1) showed a record 53.7% splicing correction following a single dose. Experts expect this fix to lead to functional benefits for patients. A minor, reversible kidney issue was noted in one patient.