Centessa provides peek at rare disease drug from extremely tiny study, leaving several questions unanswered

Max Gelman

Senior Editor

The race to develop drugs for the rare genetic disease alpha-1 antitrypsin deficiency is marching on after Vertex’s flop earlier this year, and Centessa Pharmaceuticals …

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Centessa provides peek at rare disease drug from extremely tiny study, leaving several questions unanswered

Max Gelman

Senior Editor

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Centes­sa pro­vides peek at rare dis­ease drug from ex­treme­ly tiny study, leav­ing sev­er­al ques­tions unan­swered

Max Gelman

Senior Editor

Sign up to read this article for free.

Among phar­ma CEOs, the IRA now and the IRA lat­er get very dif­fer­ent re­ac­tions

Bris­tol My­ers restarts ra­dio­phar­ma­ceu­ti­cal Phase 3 tri­al, but de­lays read­out by one year

Up­dat­ed: With cost-cut­ting un­der­way, Bris­tol My­ers rais­es guid­ance, touts Phase 3 win and scraps an­oth­er

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Centessa provides peek at rare disease drug from extremely tiny study, leaving several questions unanswered

Among pharma CEOs, the IRA now and the IRA later get very different reactions

Bristol Myers restarts radiopharmaceutical Phase 3 trial, but delays readout by one year

Updated: With cost-cutting underway, Bristol Myers raises guidance, touts Phase 3 win and scraps another

Andrew Left, short seller who campaigned against Valeant, is accused of fraud by SEC

European drug regulator rejects Alzheimer's drug Leqembi, citing safety concerns

Critical Junctures in Gene Therapy Clinical Material Manufacturing: Where Do I Start? And Will It Work?