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This week in drug discovery (13-16 May) 

Drug Discovery World

The top stories: Gene therapy cures profound genetic deafness within 24 weeks The investigational gene therapy DB-OTO improved hearing to normal levels in a child (dosed at 11 months of age) within 24 weeks, according to trial data presented at the American Society of Gene and Cell Therapy (ASGCT) annual conference.

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Spotlighting Lupus Awareness Month: CAR-T Technology Creates New Avenues for Treatment of a Devastating Disease

WCG Clinical

Recent headlines have highlighted the potential for Chimeric Antigen Receptor (CAR)-based therapies to provide clinical benefits to persons affected by lupus. Recently clinical investigators have found a way to turn what was an undesirable side effect in cancer patients into a clinical benefit for persons with certain autoimmune conditions.

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AACR: A focal point of the cancer research community

Drug Discovery World

Additionally, Ariceum Therapeutics’ Head of Translational Biology & Non-Clinical Pharmacology, Anika Jäkel, also described the conference agenda as “truly diverse,” which covered “many forms of therapeutic approaches, with some revelatory findings about new biomarkers that could lead to a new generation of personalised therapies.”

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Receptor Occupancy Assays by Flow Cytometry: Benefits for Clinical Trials

XTalks

Flow cytometric receptor occupancy assays are being increasingly used in preclinical and clinical studies. Both the areas of drug development and clinical trials are increasingly using in vitro assays to help determine the efficacy of an investigational therapeutic. What is Flow Cytometry?

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ImmunityBio’s hAd5 T-Cell COVID-19 Vaccine Candidate Shows Complete Protection of Airways in Non-Human Primates

The Pharma Data

The goal of targeting both S and N was to both activate virus-specific T cells and generate anti-SARS-CoV-2-neutralizing antibodies. The vaccine targeted both the inner nucleocapsid (N) and the outer spike (S) proteins of the virus to maximize the immune response. This can significantly limit the effectiveness of first-generation platforms.

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The challenges and trends of cell & gene therapies 

Drug Discovery World

And whilst clinical trials activity decreased by around 15% compared to 2020, last year still saw a number of regenerative medicines reach the bedside. Last year saw the sector surge, with levels of investment hitting an all-time high at $22.7 billion, compared to $19.9 billion, compared to $19.9 million price point per dose.

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Reflecting on PEGS Europe 2023 

Drug Discovery World

The company’s goal is to accelerate functional characterisation and shortenoptimisation time in antibody discovery and t-cell workflows. DDW’s Megan Thomas attended PEGS Europe 2023 in Lisbon, Portugal, from Tuesday 14 November to Thursday 16 November 2023. GSK has developed high-throughput mammalian and E.