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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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QurAlis Announces First-in-Human Dose in Phase 1 Clinical Trial of QRL-101, a First-in-Class Kv7 Precision Therapy for ALS

01/11/2023

QRL-101 aims to reduce hyperexcitability-induced motor neuron degeneration Excerpt from the Press Release: CAMBRIDGE, Mass., Jan. 5, 2023 /PRNewswire/ — QurAlis Corporation, a clinical-stage biotechnology company developing breakthrough precision medicines for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases with genetically validated targets, today announced that the company recently initiated dosing in a Phase 1…

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Surface Oncology Announces First Patient Dosed in a Phase 1/2 Study Evaluating SRF114, a Potential Best-In-Class Anti-CCR8 Antibody, in Patients with Advanced Solid Tumors

01/11/2023

SRF114 is designed to preferentially deplete CCR8+ Treg cells within the tumor microenvironment and has demonstrated highly specific CCR8-exclusive binding and reduced tumor growth in pre-clinical studies Initial clinical data anticipated in 2024 Excerpt from the Press Release: CAMBRIDGE, Mass., Jan. 05, 2023 (GLOBE NEWSWIRE) — Surface Oncology (Nasdaq: SURF), a clinical-stage immuno-oncology company developing…

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TransCode Therapeutics Announces FDA Authorization to Proceed with First-In-Human Clinical Trial

01/10/2023

Excerpt from the Press Release: BOSTON, Dec. 29, 2022 (GLOBE NEWSWIRE) — TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA Oncology Company™ committed to more effectively treating cancer using RNA therapeutics, announced today that it has received written authorization from the U.S. Food and Drug Administration (FDA) that it may proceed with its First-in-Human (FIH) Phase…

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Phio Pharmaceuticals Announces New Clinical Program to Study PH-762 for the Treatment of Cutaneous Squamous Cell Carcinoma

01/10/2023

Excerpt from the Press Release: MARLBOROUGH, Mass., Dec. 21, 2022 /PRNewswire/ — Phio Pharmaceuticals Corp., today announced it expects to file an IND in the US in the first half of 2023 for a Phase 1b clinical trial of its INTASYL™ compound, PH-762. Phio is a clinical stage biotechnology company whose proprietary INTASYL self-delivering RNAi…

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Minovia Therapeutics Reports First Clinical Data Demonstrating Disease-Modifying Efficacy and Safety of Mitochondrial Augmentation Therapy in Pediatric Patients with Primary Mitochondrial Diseases

01/10/2023

First in human clinical study provides proof-of-concept for mitochondrial augmentation therapy platform, which enables use of healthy mitochondria to improve mitochondrial function and mitigate effects of large-scale mitochondrial DNA (mtDNA) deletion syndromes Study showed improved quality of life measures in children with Pearson Syndrome and Kearns-Sayre Syndrome spectrum No treatment-related adverse effects reported in study…

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Kala Pharmaceuticals Announces FDA Acceptance of IND Application for KPI-012 for the Treatment of PCED

01/09/2023

— On-track to initiate Phase 2b trial in 1Q 2023; Topline data expected in 1Q 2024 — — Received remaining $25 million investment under previously announced private placement financing — Excerpt from the Press Release: ARLINGTON, Mass., Dec. 27, 2022 (GLOBE NEWSWIRE) — Kala Pharmaceuticals, Inc. (NASDAQ:KALA), a clinical-stage biopharmaceutical company dedicated to the research, development…

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Alnylam Submits CTA Application for ALN-KHK, an Investigational RNAi Therapeutic for the Treatment of Type 2 Diabetes

01/09/2023

–Company Expects to Initiate a Phase 1/2 Study for ALN-KHK in Early 2023, with Initial Results Expected in Late 2023 – Excerpt from the Press Release: CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi (RNA interference) therapeutics company, announced today that the Company has submitted a Clinical Trial Authorization (CTA) application to Health…

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Erasca Announces First Patient Dosed in HERKULES-1 Phase 1b Trial Evaluating ERAS-007 and ERAS-601 MAPKlamp Combination in RAS/MAPK Pathway-Altered Solid Tumors

01/06/2023

Promising preliminary monotherapy data for ERK1/2 inhibitor ERAS-007 and SHP2 inhibitor ERAS-601 in advanced solid tumors, which had favorable safety, tolerability, and efficacy profiles that support combination development BRAF Class 2 and 3 alterations have no approved targeted therapies and represent up to 25% of all BRAF-driven tumors Excerpt from the Press Release: SAN DIEGO,…

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