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VTX-801 RECEIVES U.S. FDA FAST TRACK DESIGNATION FOR THE TREATMENT OF WILSON DISEASE

The Pharma Data

Food and Drug Administration (FDA) has granted Fast Track designation to VTX-801, Vivet’s clinical-stage gene therapy for the treatment of Wilson Disease – a rare, genetic disorder that reduces the ability of the liver and other tissues to regulate copper levels, causing severe hepatic damage, neurological symptoms, and potentially death.

Disease 52
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Logistics in Focus: Orchestrating the Challenges of Cell Therapy

Conversations in Drug Development Trends

By Amy Raymond, PhD, PMP, Executive Director, Therapeutic Strategy Lead, Rare Disease Cell and gene therapies (CGTs) include cutting-edge approaches that offer the hope of a healthier, happier, and better tomorrow for a wide range of patient populations.

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How direct-to-patient clinical trial models can accelerate speed-to-market

The Pharma Data

Analyzing the benefits and challenges of implementing direct-to-patient services in the clinical supply chain to help meet patient requirements, improve supply chain efficiency and accelerate speed to market. The benefits of developing a patient-centric clinical supply chain. Add bookmark.

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A Biotech Midsummer’s Madness

LifeSciVC

And importantly, breaks from treatment or treatment protocols can literally make or break trials. There is no room for a missed dose, a scheduled follow-up visit or opportunities to bring new clinical sites on board. can have a significant impact on manufacturing timelines and drug supply chains.

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MorphoSys To Present At the 39th Annual J.P. Morgan Healthcare Conference

The Pharma Data

About MorphoSys MorphoSys is a commercial-stage biopharmaceutical company dedicated to the discovery, development and commercialization of innovative therapies for patients suffering from cancer and autoimmune diseases. In 2017, Tremfya (R) , developed by Janssen Research & Development, LLC and marketed by Janssen Biotech, Inc.,

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PharmaDrug Enters Definitive Agreement for Acquisition of Sairiyo Therapeutics Inc. Who Recently Received Orphan Drug Designation from FDA for Esophageal Cancer

The Pharma Data

Sairiyo is a biotechnology company focused on repurposing and developing improved formulations of naturally derived compounds for serious, rare, and life-threatening diseases with the aim to obtain European Medicines Evaluation Agency and U.S. Food and Drug Administration (“FDA”) approval.

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Molecular Partners Doses First Cohort in Phase 1 Trial of COVID-19 DARPin(R) Therapeutic Candidate MP0420

The Pharma Data

“In collaboration with our partner, Novartis, our team continues to move at extraordinary speed from idea to bench to clinical trials, driven by a common desire to help patients against this devastating disease and help bring the world back closer to normalcy.” million doses of MP0420, if it is approved in Switzerland.

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