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PepGen’s early Phase 2 data lift shares as Ionis and ImmunityBio fall

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PepGen reported positive Phase 2 results for PGN-EDODM1, its investigational oligonucleotide for myotonic dystrophy type 1 (DM1). After four monthly doses, the six treated participants in the 10 mg/kg cohort showed average muscle RNA-splicing correction of 46.5%; four had correction above 50%. The small cohort also showed improvements on measures including hand-opening time, grip strength and mobility. The company reported no serious or severe adverse events or treatment discontinuations at this dose. These early findings need confirmation in larger, longer studies. PepGen shares rose sharply on the news. Its higher-dose 12.5 mg/kg cohort is fully enrolled, with results expected in the first half of 2027. The study remains subject to a U.S. FDA partial clinical hold following nonclinical findings in mice. Ionis shares fell after the company cut its sales outlook for Tryngolza, while ImmunityBio shares sagged after quarterly revenue missed estimates despite reporting positive blad...

Beam reports positive Phase 1/2 results for base-edited sickle cell therapy

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Democrats step up scrutiny of confidential MFN drug deals

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aTyr Pharma files to sell up to $80 million in stock

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iORGANBIO and Mitsui Chemicals team up on AI-driven cell and organoid manufacturing

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FDA investigates tumor reports linked to Zolgensma

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argenx to acquire Unnatural Products for up to $1.15 billion

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Hims & Hers and Eli Lilly agree to offer branded weight-loss drugs

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Novartis’s Lp(a) drug sharply lowered the biomarker but did not prevent cardiovascular events

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J&J’s oral Icotyde shows promising PsA results, but higher dose misses statistical mark

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Zura Bio drops HS program after Phase 2 trial miss

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CRISPR reports encouraging early zugo-cel responses across autoimmune disease cohorts

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Vir’s hepatitis D drug combination succeeds in first pivotal study

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