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Start-up aims to advance transformative genetic medicines

Drug Discovery World

Challenges remain with current delivery approaches, and genetic medicines applications have been generally restricted to the liver and ex vivo settings. Aera’s technology also includes a licensed therapeutic enzyme platform based on the discovery of novel, compact, and programmable gene editing enzymes.

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MiNA and BioMarin partner to speed development of RNAa therapies

Pharmaceutical Technology

MiNA Therapeutics has entered into a research collaboration and option licensing agreement with BioMarin Pharmaceutical to speed up the development of therapeutic ribonucleic acid activation (RNAa) candidates to treat rare genetic diseases. The option licensing agreement is based on early-stage clinical results.

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This week in drug discovery (2-6 October)  

Drug Discovery World

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

Drugs 52
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Bayer to acquire exclusive license from Cedilla Therapeutics on selective inhibitors in pre-clinical precision oncology

The Pharma Data

Overexpression or genetic activation of Cyclin Dependent Kinase 2 (CDK2) binding partner cyclin E is a key oncogenic process in several cancers. Cedilla’s small molecules conditionally modulate the protein complex in its functional state resulting in highly selective inhibition. Financial details have not been disclosed.

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Beqvez Becomes Pfizer’s First Approved Gene Therapy After Nod from Health Canada

XTalks

Hemophilia B, also known as congenital Factor IX (FIX) deficiency or Christmas disease, is a rare genetic bleeding disorder caused by a deficiency or defect in the FIX clotting protein. Treatment involves replacing the missing FIX clotting protein, traditionally through regular infusions. Priced at $3.5

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RNA editing oligonucleotide selected for AATD treatment 

Drug Discovery World

Korro’s RNA editing approach has the potential to be transformative for AATD patients and Korro is well-positioned to deliver a best-in-class therapeutic.

RNA 52
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GSK pumps £30m into a new Oxford University partnership

pharmaphorum

GlaxoSmithKline has formed a five-year partnership with Oxford University to set up a new institute that will apply machine learning and functional genomics to the discovery of new medicines. Genetics, proteomics and digital pathology will be harnessed in order to understand the patterns of disease which vary amongst individuals, said GSK.