Remove Genetic Disease Remove Licensing Remove Life Science
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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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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.

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Rare diseases, repurposing and the role of AI

pharmaphorum

These might be licensed drugs that could hold potential for a patent extension, or drugs which failed efficacy trials for an intended indication. Findacure is a charity that works directly with rare disease patient groups to help them grow and professionalise. The post Rare diseases, repurposing and the role of AI appeared first on.

Trials 105
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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
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Acadia closer to bringing first Rett syndrome drug to market

pharmaphorum

If approved, the drug will be sold by Neuren Pharma, which licensed North American rights to the drug in 2018 and is also developing another drug for Rett blarcamesine, in collaboration with Anavex Life Sciences. Results from a phase 3 trial of blarcamesine are due next year.

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Biopharma Money on the Move: December 2 – 8

The Pharma Data

December is bringing frost in the North and plenty of cold hard cash for these life sciences companies. . European venture capital firm Forbion rounded up $545 million for its fifth life sciences fund. The impact of CNS diseases extends beyond patients—to their families and society as well.”

RNA 52
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BioSpace Movers & Shakers, Oct. 16

The Pharma Data

Sabbagh will be responsible for expanding Inozyme’s proprietary pipeline by identifying and developing new therapeutics for monogenic and non-genetic diseases of abnormal mineralization. Prior to joining Inozyme, Sabbagh served as the head of rare renal and musculoskeletal diseases research at Sanofi.

In-Vivo 52