XTalks

DECEMBER 4, 2024

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.

Bioavailability 111

Bioavailability Genetic Disease Marketing RNA 111

XTalks

DECEMBER 18, 2024

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

Genetics Hormones Trials Drugs 116

pharmaphorum

OCTOBER 16, 2020

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

Trials Clinical Trials Clinical Trials Life Science 105

XTalks

SEPTEMBER 13, 2021

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

RNA Genetics Genetic Disease Medicine 105

pharmaphorum

DECEMBER 8, 2021

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.

Marketing 59

Marketing Drugs Life Science Trials 59

The Pharma Data

DECEMBER 8, 2020

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

RNA Antibody Life Science Protein 52

The Pharma Data

OCTOBER 15, 2020

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

In-Vivo Business Development Manufacturing Development 52