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Regulator and Funder? FDA’s Orphan Products Grants Program awards significant funding to help move promising treatments through clinical development

FDA Law Blog: Drug Discovery

The Clinical Trials Grants Program provides funding for clinical trials that evaluate the safety and efficacy of potential treatments for rare diseases to help move promising treatments through clinical development. This study design is both innovative and efficient, leveraging a single-arm, baseline-controlled design.

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FDA approves Phase 1 trial for HIV gene therapy

The Pharma Data

The FDA has approved a request from American Gene Technologies to begin a clinical study into its HIV gene therapy. In a paper published in Molecular Therapy: Methods & Clinical Development , the team discussed why they choose this type of gene therapy to treat HIV. Conor Kavanagh.

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Where is AI winning in drug discovery? 4 use cases to know

Fierce BioTech

Even though AI-designed drugs arent yet a household term for FDA-approved, commercially available therapies, they are a reality in clinical development pipelines.

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FDA Approves Updated Indication for Merck’s KEYTRUDA® (pembrolizumab) for Treatment.

The Pharma Data

today announced a label update for KEYTRUDA, Merck’s anti-PD-1 therapy, for its indication in first-line advanced urothelial carcinoma (bladder cancer) in the U.S. Food and Drug Administration (FDA) has converted this indication from an accelerated to a full (regular) approval. Source link: [link].

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The science of ageing and restoring healthspan

Drug Target Review

Contrary to popular belief, ageing is not caused by just random wear and tear of our bodies over time but is instead caused by a discrete set of biological mechanisms that we now better understand and can target with therapies. a clinical-stage private biopharma company developing therapies for neurological and psychiatric diseases.

Science 145
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FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1

The Pharma Data

FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1. Food and Drug Administration (FDA) approved Oxlumo (lumasiran) injection for subcutaneous use, the first-ever therapy available for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.

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A research team searches for every gene that helps tumors evade immunotherapy

Broad Institute

But immunotherapy was not yet widely used and had not been applied clinically to Merkel cell carcinoma, so she received traditional chemotherapy and radiation therapy, suffering life-threatening complications along the way. His mother had a presentation of the disease that suggested her immune system was already on the job.

Research 137