太平洋生物科学公司将与基金会合作研究罕见遗传疾病疗法

2026-01-13 00:23

Pacific Biosciences of California (PACB) will work with the n-Lorem and EspeRare Foundations to use long-read whole-genome sequencing to develop personalized antisense oligonucleotide therapies for ultra-rare genetic diseases.
By integrating the sequencing earlier in development, "the proposed collaboration will aim to reduce the need for multiple sequential tests, accelerate target validation, and improve confidence in ASO design."
PacBio will donate sequencing reagents and offer scientific resources to support long-read genome analysis for research in support of target identification, ASO design feasibility, and regulatory-grade molecular characterization.

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