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#核酸药物

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Oct 14Wed
  1. biospace.com48

    Alnylam bulls and bears fight it out after business-shaking guidance cut

    Alnylam cut full-year guidance by about $200 million after second-quarter sales of the TTR silencer Amvuttra in transthyretin-mediated amyloidosis fell short, and its stock has dropped from near $500 to around $250. Analysts at Guggenheim question whether the ATTR-CM opportunity is smaller than expected or whether stabilizers from Pfizer and BridgeBio are becoming preferred, while TD Cowen reiterated a $482 target and BMO argues the cut reflects second-line patient exhaustion. Alnylam is running Phase 3 trials of nucresiran in ATTR-CM and ATTR-PN, and a Phase 3 cardiovascular outcomes trial of zilebesiran in hypertension.

Sep 2Wed
  1. fiercebiotech.com48

    Is Moderna, Merck's mRNA cancer vaccine win the start of a 'personalized medicine revolution'?

    Merck & Co. and Moderna reported in August that their individualized mRNA cancer vaccine intismeran autogene (V940, mRNA-4157) plus Keytruda significantly improved recurrence-free survival and distant metastasis-free survival versus Keytruda alone in the phase 3 INTerpath-001 trial of 1,137 patients with resected cutaneous melanoma. Detailed data are being held for a future medical meeting, and analysts and academics say the result has revived interest in mRNA-based personalized cancer vaccines. Moderna's stock closed up 177% on the news.

Aug 21Fri
  1. biocentury.com56

    Mapping first steps toward scaling genetic medicines: C-Path’s Romero on the BioCentury Show

    The Critical Path Institute's One to Millions Initiative is building a development framework for individualized rare disease therapies, starting with antisense oligonucleotides, and aims to extend it to all genetically defined diseases, including mutation-segmented subtypes of Parkinson's disease. CEO and CSO Klaus Romero said the bottleneck is the absence of a standardized regulatory and development system, and the initiative's first deliverables include a shared safety database meant to reduce repeated preclinical studies. The effort implements FDA's Plausible Mechanism Framework through consortia of companies, academics, patient groups and regulators sharing data.

CBACBA Frontier Insights (CFI)Chinese Biopharmaceutical Association (CBA)www.cba-usa.org