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Arrowhead and Sarepta link for rare genetic disease treatments

Pharmaceutical Technology

Arrowhead has entered a worldwide licensing and partnership agreement with Sarepta Therapeutics for rare genetic disease treatments.

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Jupiter Neurosciences Makes Way in CNS Treatments with $11M IPO

XTalks

These efforts aim to fast-track commercialization and out-licensing agreements in one of the world’s most promising pharmaceutical markets. CAMP4’s RNA-based therapies focus on genetic diseases like urea cycle disorders, while Rapport’s small molecules aim to address epilepsy, pain and bipolar disorder.

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Astellas to support development of Taysha’s gene therapy programmes

Pharmaceutical Technology

Astellas chief strategy officer Naoki Okamura said: “Gene therapy is the cornerstone of Astellas’ Primary Focus, Genetic Regulation; our goal is to bring new transformative treatment options to patients living with serious genetic diseases and limited treatment options.

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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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Astellas makes another gene therapy play, takes stake in Taysha

pharmaphorum

The Japanese firm has agreed to make a $50 million investment in Dallas-based Taysha in exchange for a 15% stake in the company, plus exclusive options to license two clinical-stage, single-gene therapies for rare genetic diseases.

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Neurocrine Biosciences’ Crenessity Approved as First New Treatment in Decades for Rare Genetic Disorder CAH

XTalks

In 2018, the company also received approval for Orilissa (elagolix), which it licenses to AbbVie, for the treatment of endometriosis. It also received the FDAs rare pediatric disease priority review voucher along with the approval, which will help expedite the agencys assessment of another application. billion in revenue by 2024.

Genetics 116
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Eli Lilly Pursues RNA Editing in New Partnership with ProQR

XTalks

With RNA therapies being the next hot thing in genetic medicine, Eli Lilly is joining the RNA editing race by partnering with Netherlands-based ProQR Therapeutics NV (Nasdaq: PRQR), a biotech company developing RNA-based therapies for rare genetic diseases with a focus on blinding disorders of the retina.

RNA 105