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First Positive Data of In Vivo CRISPR-Based Genome Editing in Humans Reported

First Positive Data of In Vivo CRISPR-Based Genome Editing in Humans Reported June 28, 2021 A team of researchers based in the U.S., the U.K., and New Zealand from Intellia Therapeutics, Regeneron Pharmaceuticals, and clinical partners showed in a study that the companies’ lead in vivo genome editing candidate NTLA-2001 generated a dose-dependent sustained reduction of protein linked to transthyretin (ATTR) amyloidosis following a single dose in six patients living with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN). The study is the first ever to support the safety and efficacy of in vivo CRISPR genome editing in humans. The New England Journal of Medicine and in a presentation that day at the 2021 Peripheral Nerve Society (PNS) Annual Meeting the researchers reported that a single 0.3 mg/kg dose of NTLA-2001 led to an 87% mean reduction in serum transthyretin (TTR) protein concentration in three of the ATTRv-PN patients by day 28, with individual re

New Era of Medicine : Researchers Publish First Positive Clinical Data for In Vivo Genome Editing in Humans

New Era of Medicine : Researchers Publish First Positive Clinical Data for In Vivo Genome Editing in Humans
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Ionis antisense therapies in ATTR amyloidosis to be featured at 2021 PNS Annual Meeting

Emerging Therapeutics to boom AL Amyloidosis Treatment Synopsis

What are the current options for Amyloid light-chain Amyloidosis treatment? How many companies are developing therapies for the treatment of AL Amyloidosis?  How many are Amyloid light-chain Amyloidosis emerging therapies in the early-stage, mid-stage, and late stages of development for the treatment of AL Amyloidosis? What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, and significant licensing activities that will impact the Amyloid light-chain Amyloidosis market?  Which are the dormant and discontinued products and the reasons for the same? What is the unmet need for current therapies for the treatment of AL Amyloidosis?   What are the current novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitation of existing Amyloid light-chain Amyloidosis therapies? 

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