STAT News

NOVEMBER 14, 2022

Ionis Pharmaceuticals, the California company that turned RNA-targeting medicines from an unproven idea into blockbuster drugs, is now expanding into a new class of therapeutics that treat disease by editing DNA.    On Monday, the company announced a partnership with Metagenomi to kickstart these efforts.

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Bio Pharma Dive

NOVEMBER 14, 2022

The RNA drug developer is paying the richly-funded startup $80 million in a wide-ranging alliance that marks its first foray into DNA editing medicines.

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Gene Editing RNA DNA Gene 161

Camargo

JULY 27, 2021

When developing a clinical development program for these increasingly popular therapies, it is important that sponsors use modeling and pharmacokinetic (PK) analysis to evaluate parameters that can be measured while dosing with gene therapy drugs, to characterize exposure-response data and inform rational dosing.

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The Pharma Data

JUNE 7, 2023

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., LNPs are spheric drug delivery bodies which can be equipped with therapeutic payloads for intracellular delivery. Financial details were not disclosed. Source link: [link]

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XTalks

NOVEMBER 30, 2023

In 2022, the US Food and Drug Administration (FDA) granted approval to the medication Sunlenca (lenacapavir) for adults living with multi-drug resistant HIV-1 infection. Developed by Gilead, Sunlenca is the first drug in a novel class known as capsid inhibitors to receive FDA approval for treating HIV-1.

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FDA Approval Gene Therapy Gene Clinical Trials 110

Delveinsight

FEBRUARY 25, 2021

Beam adds to drug delivery stable with USD 120 Million GuideTx buy. The gene-editing technologies, which have reached the clinic, CRISPR, zinc finger nucleases, and TALENs, make their edits by nicking DNA at the target site. GRAIL and Quest collaborate for the cancer blood test.

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DNA In-Vivo Genetics Medicine 52

The Pharma Data

DECEMBER 5, 2020

David Williams, uses a virus to introduce RNA into the extracted bone marrow that turns off BCL11A. The other study, from German researchers led by Dr. Selim Corbacioglu from the University of Regensburg, relies on CRISPR-Cas9 gene editing to switch off BCL11A. Food and Drug Administration.

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Gene Therapy Gene Gene Editing Genetics 52