Gene Therapy

Integral Molecular Reveals Mechanism Underlying Exquisite Specificity of Claudin 6 Therapeutic Antibody Being Developed for Solid Tumors

01/13/2023

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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Orchard Therapeutics Announces U.S. FDA Clearance of IND Application for OTL-203 in MPS-IH

01/12/2023

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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Homology Medicines Provides Update on pheEDIT and juMPStart Clinical Trials and Announces Expected 2023 Milestones, Including Initial Data Read-Outs from Both Programs

01/11/2023

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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Spirita Oncology, LLC Initiates a Phase 1 Clinical Trial of the Anti-Cancer Agent E6201 in Combination with Dabrafenib in Patients with Central Nervous System Metastases from BRAF V600 Mutated Metastatic Melanoma

12/08/2022

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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Invitae Launches Rare Patient Network for Pediatric Patients With Rare Neurodevelopmental Diseases

12/07/2022

– 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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Lineage Announces Launch of Phase 2a Study by Genentech of RG6501 (OpRegen®) in Patients With Geographic Atrophy Secondary to Age-Related Macular Degeneration

11/29/2022

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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World’s largest autism whole genome sequencing study reveals 134 autism-linked genes

11/25/2022

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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Escient Pharmaceuticals Initiates Phase 2 PACIFIC Study Evaluating EP547, An Oral MRGPRX4 Antagonist, in Subjects with Cholestatic Pruritus

11/24/2022

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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4D Molecular Therapeutics Announces Interim Clinical Data from Phase 1/2 Clinical Trial of 4D-710 for Cystic Fibrosis Lung Disease at NACFC 2022

11/10/2022

Cohort 1 lung bronchoscopy sample results demonstrate widespread delivery and expression of the 4D-710 CFTR∆R transgene in 100% of samples from all three patients Cohort 1 safety and tolerability of 4D-710 demonstrated to date, with no drug-related adverse events following aerosol delivery Cohort 1 enrollment completed; Cohort 2 enrollment on-going Conference call & webcast to…

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T2 Biosystems Initiates Studies to Add Acinetobacter Baumannii to the FDA-Cleared T2Bacteria Panel

10/28/2022

Excerpt from the Press Release: LEXINGTON, Mass., Oct. 20, 2022 (GLOBE NEWSWIRE) — T2 Biosystems, Inc. (NASDAQ:TTOO), a leader in the rapid detection of sepsis-causing pathogens and antibiotic resistance genes, announced today that it has initiated studies to expand the number of pathogens detected on the FDA-cleared T2Bacteria® Panel to include the detection of Acinetobacter baumannii.…

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