Gene Therapy
Excerpt from the Press Release: OAKLAND, Calif., Jan. 4, 2023 /PRNewswire/ — Tempero Bio, Inc, a clinical-stage biopharmaceutical company focused on developing transformative therapies for the treatment and prevention of alcohol and other substance use disorders, today announced the U.S. Food and Drug Administration (FDA) clearance of its Investigational New Drug (IND) application for TMP-301.…
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As previously reported, resmetirom demonstrated improvements in NASH and liver fibrosis on liver biopsies, the primary endpoints of the MAESTRO-NASH trialA supportive analysis using consensus reads of digitized biopsy images by the central pathologists replicated the positive primary endpoint resultsThese data will be presented at the NASH-TAG Conference on Friday January 6th, 2023 Excerpt from…
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Excerpt from the Press Release: PHILADELPHIA–Integral Molecular, the industry leader in antibody discovery for membrane proteins, has published the targeting mechanism enabling best-in-class specificity of its Claudin 6 (CLDN6) antibody CTIM-76 being developed for cancer therapy with Context Therapeutics. CLDN6 is a tumor-specific protein found in multiple solid tumors—including ovarian, endometrial, lung, gastric, and testicular—but…
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Global registrational trial expected to commence in the second half of 2023 Excerpt from the Press Release: BOSTON and LONDON, Jan. 05, 2023 (GLOBE NEWSWIRE) — Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for OTL-203, a…
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Strong Cash Position with Runway into Fourth Quarter 2024 Non-Clinical Data on Immunosuppression Regimen Supportive of Clinical Programs Preclinical Data from HMI-103 Gene Editing Program Utilizing a Unique Mechanism of Action Demonstrated Significantly Increased Potency in PKU Model Excerpt from the Press Release: EDFORD, Mass., Jan. 04, 2023 (GLOBE NEWSWIRE) — Homology Medicines, Inc. (Nasdaq:…
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Excerpt from the Press Release: BOSTON–(BUSINESS WIRE)–Spirita Oncology, LLC, has initiated a Phase 1 clinical trial of anti-cancer agent E6201 in combination with dabrafenib in patients with BRAF V600-mutated metastatic melanoma that has spread to the central nervous system. There are approximately 47,000 new cases of metastatic melanoma in the U.S. each year, with approximately…
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– Ciitizen real-world data platform expansion helps advance research and potentially improve outcomes for patients with pediatric epilepsy and/or developmental delay – Excerpt from the Press Release: SAN FRANCISCO, Dec. 1, 2022 /PRNewswire/ — Invitae (NYSE: NVTA), a leading medical genetics company, today announced the launch of the Rare Patient Network expanding Invitae’s Ciitizen platform to…
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Excerpt from the Press Release: CARLSBAD, Calif.–(BUSINESS WIRE)–Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, announced today that its partner Genentech, a member of the Roche Group, has launched a Phase 2a, multicenter, open-label, single arm clinical study of RG6501 (OpRegen), a…
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Summary: SickKids-led study sequences the entire genomes of more than 11,000 individuals, offering new insights into the genetics that underlie autism spectrum disorder. Excerpt from the Press Release: Researchers from The Hospital for Sick Children (SickKids) have uncovered new genes and genetic changes associated with autism spectrum disorder (ASD) in the largest autism whole genome…
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Excerpt from the Press Release: SAN DIEGO–(BUSINESS WIRE)–Escient Pharmaceuticals, a clinical-stage company advancing novel small molecule therapeutics for the potential treatment of a broad range of neurosensory-inflammatory disorders, today announced the initiation of a Phase 2 clinical study of EP547 in subjects with cholestatic pruritus. EP547 is a highly selective antagonist of MRGPRX4, a cell…
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