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A Visual Guide to Genome Editors

Codon

The treatment, now known as Casgevy, became the first CRISPR-based therapy to gain FDA approval, in 2023. The core of the CRISPR immune response is a guide RNA (gRNA) that binds to a CRISPR-associated (Cas) protein. Collectively, these repeat-protospacer regions are known as CRISPR arrays. Visit our website to get a copy.

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Levers for Biological Progress

Codon

Scientists are already building a model that can, for example, look at which RNA molecules are expressed in a cell at t=0 and predict how those molecules will change at t=1. SeqFISH, developed by Long Cai’s group at Caltech, maps the spatial positions of thousands of RNA, DNA, or protein molecules within single cells.

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New gene delivery vehicle shows promise for human brain gene therapy

Broad Institute

By Allessandra DiCorato May 16, 2024 Credit: Deverman lab Brain vasculature (in blue) surrounded by RNA (in orange) transcribed from the gene delivered to the brain in humanized mice using an engineered AAV targeting the human transferrin receptor.

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Biopharma Money on the Move: December 2 – 8

The Pharma Data

Laying down a new track for RNA processing, Remix launched with $81 million in financing. Funds will be used to support development of the REMaster technology platform and advance the company’s pipeline of RNA processing targeted therapeutics as well. “As Pear Therapeutics. Most read today on BioSpace: Source link.

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Janssen Announces U.S. FDA Approval of CABENUVA (rilpivirine and cabotegravir), the First Long-Acting Regimen for the Treatment of HIV

The Pharma Data

CABENUVA, a co-packaged kit with two injectable medicines, offers people living with HIV a new approach for maintaining viral suppression. Click to Tweet : #BREAKING: The #FDA has approved another treatment option for people living with #HIV. Two percent of patients in both treatment arms had an HIV-1 RNA count ?50

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Months of Work Hang in the Balance as Moderna’s COVID-19 Vaccine Awaits AdComm Vote

The Pharma Data

To this point, Moderna has only submitted two months of follow-up safety data, and the FDA typically requires six months for a full approval. intends to ship just shy of six million doses of Moderna’s vaccine once the FDA approves EUA. Initial doses are expected to be limited as manufacturing ramps up.

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Codon Digest: Injected Gene Editors

Codon

Read A new DNA polymerase variant, called RT-KTq I614Y, can directly detect RNA modifications, including pseudouridine (Ψ) and queuosine (Q). By combining this new variant with standard sequencing methods, it’s possible to identify RNA modifications in a really simple way. Nucleic Acids Research. Nature Biotechnology.

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