gene therapy

Jasper Therapeutics Announces New Positive Briquilimab Data to be Presented at the 2023 Tandem Meetings: Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR


Three abstracts highlight the safety and efficacy of briquilimab combined with low-toxicity radiation conditioning to achieve full donor engraftment and leukemia disease eradication Excerpt from the Press Release: REDWOOD CITY, Calif., Jan. 13, 2023 (GLOBE NEWSWIRE) — Jasper Therapeutics, Inc. (Nasdaq: JSPR) (“Jasper” or the “Company”), a biotechnology company developing novel antibody therapies addressing chronic…

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Tempero Bio Announces FDA Clearance of Investigational New Drug (IND) Application for TMP-301 for the Treatment of Alcohol and Other Substance Use Disorders


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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Madrigal Announces Additional Positive Results from the Pivotal Phase 3 MAESTRO-NASH Clinical Trial of Resmetirom for the Treatment of NASH with Liver Fibrosis


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


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


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


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


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


– 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


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


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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