Industry News

Editas Medicine Presents Preclinical Data on EDIT-103 for Rhodopsin-associated Autosomal Dominant Retinitis Pigmentosa at the Association for Research in Vison and Ophthalmology Conference

05/12/2022

Studies in non-human primates demonstrated nearly 100% gene editing knockout of endogenous RHO gene and more than 30% replacement protein levels Treated eyes showed morphological and functional photoreceptor preservation  EDIT-103 advancing towards IND-enabling studies Excerpt from the Press Release: CAMBRIDGE, Mass., May 04, 2022 (GLOBE NEWSWIRE) — Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome…

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Gamida Cell Presents New Data from NAM-Enabled Genetically Modified Natural Killer (NK) Pipeline at International Society for Cell & Gene Therapy 2022

05/11/2022

Poster selected for inclusion in conference’s Elevator Pitch Session: GDA-301 produces enhanced potency and persistence with combined genetic manipulation of CISH gene editing and the engineered expression of membrane-bound IL-15 for targeting hematologic malignancies and solid tumors GDA-601 generates promising immunotherapeutic potential to target multiple myeloma cells Company plans to select a genetically modified NK…

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Kymera Therapeutics to Share Novel Preclinical Findings Reinforcing the Advantage of IRAK4 Degraders over Kinase Inhibitors as well as First STAT3 Degrader Results in a Preclinical Model of Th17 Inflammation

05/11/2022

-Data to be shared at the American Association of Immunologists (AAI) Annual Meeting -Results highlight potential for IRAK4 degraders to broadly impact TLR/IL-1R-driven inflammatory and autoimmune diseases in a manner superior to kinase inhibitors -Findings support further exploration of STAT3 degraders in Th17-driven autoimmune indications Excerpt from the Press Release: WATERTOWN, Mass., May 05, 2022…

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Marinus Pharmaceuticals Announces Resumption of Phase 3 RAISE Trial in Status Epilepticus

05/11/2022

Excerpt from the Press Release: RADNOR, Pa.–(BUSINESS WIRE)–Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today announced that it has resumed screening and recruitment for the Phase 3 RAISE (Randomized Therapy in Status Epilepticus) trial in refractory status epilepticus (RSE). The company continues to…

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KVK Tech Announces Phase II Trial of COVID-19 Oral Therapy is Currently Enrolling in Nepal

05/10/2022

Excerpt from the Press Release: NEWTOWN, Pa., May 5, 2022 /PRNewswire/ — Sen-Jam Pharmaceuticals and KVK Tech are collaborating to develop a Covid-19 oral therapy. The companies will operate in parallel while they complete the Phase II clinical trials in Nepal. KVK Tech will offer formulation and chemistry, manufacturing and controls (CMC) for the oral therapy. Furthermore, KVK…

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Phio Pharmaceuticals Announces the Start of its Phase 1b Clinical Trial of PH-762 for the Treatment of Advanced Melanoma

05/10/2022

Excerpt from the Press Release: MARLBOROUGH, Mass., April 26, 2022 /PRNewswire/ — Phio Pharmaceuticals Corp. (Nasdaq: PHIO), a clinical stage biotechnology company developing the next generation of therapeutics based on its proprietary self-delivering RNAi (INTASYL™) therapeutic platform, announced today that it has opened patient enrollment in the Phase 1b clinical trial of PH-762 for the treatment of advanced melanoma. Logo…

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Charles River and Valo Launch Logica, an Integrated AI-Powered Drug Discovery Solution to Rapidly Deliver Optimized Preclinical Assets

05/10/2022

Customized drug discovery offers integrated target-to-lead and target-to-clinic ready small molecules Excerpt from the Press Release: WILMINGTON, Mass. & BOSTON–(BUSINESS WIRE)–Charles River Laboratories International, Inc. (NYSE: CRL) and Valo Health, Inc (“Valo”) have launched LogicaTM, an artificial intelligence (AI) powered drug solution that directly translates clients’ biological insights into optimized preclinical assets. Logica leverages Valo’s…

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HARMONY BIOSCIENCES INITIATES PHASE 3 TRIAL OF PITOLISANT IN ADULT PATIENTS WITH IDIOPATHIC HYPERSOMNIA

05/09/2022

Excerpt from the Press Release: PLYMOUTH MEETING, PA, April 27, 2022— Harmony Biosciences Holdings, Inc. (“Harmony”) (Nasdaq: HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients with rare neurological diseases, today announced the initiation of its Phase 3 trial evaluating the efficacy and safety of pitolisant in adult patients with idiopathic hypersomnia…

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ChristianaCare Scientists Show for the First Time That Tumor Cells Can Manipulate the Body’s Natural Antibody Response to Triple Negative Breast Cancer

05/09/2022

Findings point to potential new therapeutic targets for this highly aggressive, drug-resistant breast cancer subtype Excerpt from the Press Release: In breakthrough research at ChristianaCare’s Helen F. Graham Cancer Center & Research Institute, scientists have discovered that a protein secreted by tumor cells can switch off the body’s natural defenses against triple negative breast cancer (TNBC).…

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Preclinical Research Published in Science Advances Demonstrates Repertoire Immune Medicines’ Cell-Tethering Technology Delivers Potent Interleukin-12 Directly to Solid Tumors Potentially Improving Effectiveness of Cellular Immunotherapies

05/09/2022

Repertoire’s proprietary cell-tethering technology limits systemic toxicity typically associated with use of immunomodulatory IL-12 and allows direct delivery to solid tumors in mouse models Tethered IL-12 repolarizes the tumor microenvironment potentially creating improved conditions for cellular immunotherapies to work effectively Excerpt from the Press Release: CAMBRIDGE, Mass.–(BUSINESS WIRE)–Repertoire Immune Medicines announced today that Science Advances published preclinical research…

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