Remove tag next-generation-drug-delivery
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First gene therapy trial for Hunter syndrome opens in Manchester, UK

Drug Discovery World

The drug was developed over eight years by Brian Bigger, Professor of Cell and Gene Therapy at The University of Manchester. Next generation stem cell gene therapy Children with Hunter syndrome have a missing gene, meaning they cannot produce an important enzyme called iduronate-2-sulfatase or IDS.

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Grand Rounds September 16, 2022: Using Nationwide Registries to Conduct Pragmatic Randomized Trials: The DANFLU Program (Tor Biering-Sørensen, MD, PhD, MPH)

Rethinking Clinical Trials

The system is used for priority communications from government agencies, hospitals, banks, and others, and allows for rapid delivery of recruitment letters and implementation interventions. The system is used by approximately 5.8 million people, representing more than 95% of the population. Learn more. Read about DANFLU-1.

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Amtagvi (Lifileucel) Scores Landmark Win as First FDA-Approved T-Cell Therapy for a Solid Tumor

XTalks

Iovance Biotherapeutics’ Amtagvi (lifileucel) won US Food and Drug Administration (FDA) approval last week for the treatment of advanced melanoma, making it the first individualized tumor-infiltrating lymphocyte (TIL) therapy and the first T-cell therapy for a solid tumor to win US regulatory approval.

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What to expect from PEGS Europe 2023: Day 3

Drug Discovery World

On Day 3, Thursday 16 November 2023, the tracks include: the second part of machine learning for protein engineering, antibodies against membrane protein targets, engineering bispecific antibodies, next-generation immunotherapies, protein stability and formulation, and protein process development.

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What to expect from PEGS Europe 2023: Day 2

Drug Discovery World

The first three include: Sungjin Park, PhD, CEO, Onegene Biotechnology, on: ‘UniStac: Enzyme-mediated conjugation technology for accelerated development of tetraspecific NASH drug’. Pawel Stocki, PhD, Vice President Research, Ossianix, on: ‘Advancing brain shuttle-enabled therapeutics for efficient delivery to cns —translation to primates’.

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PFIZER REPORTS STRONG FIRST-QUARTER 2021 RESULTS

The Pharma Data

Food and Drug Administration (FDA) approved the supplemental New Drug Application (sNDA) for Lorbrena, expanding the indication to include first-line treatment of people with anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC). Lorbrena (lorlatinib) — In March 2021, Pfizer announced that the U.S.