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U.S. Food and Drug Administration approves CSL’s HEMGENIX® (etranacogene dezaparvovec-drlb), the first gene therapy for hemophilia B

12/01/2022

KING OF PRUSSIA, PA, USA This historic approval provides a new treatment option that reduces the rate of annual bleeds, reduces or eliminates the need for prophylactic therapy and generates elevated and sustained factor IX levels for years after a one-time infusion. Excerpt from the Press Release: This historic approval provides a new treatment option…

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Zynerba Pharmaceuticals Announces Publication of Data from Phase 3 CONNECT-FX Study of Zygel™ in the Journal of Neurodevelopmental Disorders

12/01/2022

Study finds patients with Fragile X who have a highly methylated FMR1 gene that were treated with Zygel showed a significant reduction in behavioral symptoms compared to those treated with placebo Topline results from follow-on RECONNECT confirmatory pivotal Phase 3 trial of Zygel in patients with a completely methylated FMR1 gene expected in second half…

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Nabriva Therapeutics Announces Positive Topline Results from Phase 1 Trial of XENLETA® (lefamulin) in Adult Patients with Cystic Fibrosis

12/01/2022

– PK Data and Safety Profile Consistent with Previous Studies in Healthy Subjects – – Results Support Potential Utility of Lefamulin in Patients with Cystic Fibrosis – Excerpt from the Press Release: DUBLIN, Ireland and FORT WASHINGTON, Pa., Nov. 28, 2022 (GLOBE NEWSWIRE) — Nabriva Therapeutics plc (NASDAQ: NBRV), a biopharmaceutical company engaged in the…

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Immuneering Announces First Patient Dosed in its Phase 1/2a Clinical Trial of IMM-1-104 in Advanced Solid Tumors with RAS Mutations

11/30/2022

IMM-1-104 being tested in first all-comers RAS clinical trial believed to be conducted to date First patient dosed follows swiftly from IND clearance at the end of Septembe Excerpt from the Press Release: CAMBRIDGE, Mass., Nov. 28, 2022 (GLOBE NEWSWIRE) — Immuneering Corporation (Nasdaq: IMRX), a clinical stage biopharmaceutical company that aims to create medicines…

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Morphic Announces Initiation of EMERALD-2 Phase 2b Clinical Trial of MORF-057 in Patients with Moderate-to-Severe Ulcerative Colitis

11/30/2022

First patient dosed in global study of oral α4β7 integrin inhibitor Excerpt from the Press Release: WALTHAM, Mass., Nov. 22, 2022 (GLOBE NEWSWIRE) — Morphic Therapeutic (Nasdaq: MORF), a biopharmaceutical company developing a new generation of oral integrin therapies for the treatment of serious chronic diseases, today announced that the EMERALD-2 phase 2b study of…

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Praxis Precision Medicines to Advance PRAX-562 Phase 2 Study in Pediatric Patients with Developmental and Epileptic Encephalopathies

11/30/2022

Excerpt from the Press Release: BOSTON, Nov. 28, 2022 (GLOBE NEWSWIRE) — Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today announced plans to initiate the PRAX-562 Phase 2 EMBOLD study for the treatment of…

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Biological Dynamics collaborates on clinical utility studies to examine the impact of its early pancreatic cancer detection assay

11/29/2022

The company partners with QURE and Curta to assess value of early identification in high-risk cohorts Excerpt from the Press Release: SAN DIEGO, Nov. 22, 2022 /PRNewswire/ — Biological Dynamics, an exosome-isolation technology and early disease detection diagnostic company, today announced that it has partnered with both QURE Healthcare and Curta to explore how the…

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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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Unexpected Result: COVID-19 Vaccination Improves Effectiveness of Cancer Treatment

11/29/2022

Excerpt from the Press Release: Clinical study on nasopharyngeal cancer with an unexpected result. Patients with nasopharyngeal cancer are often treated with drugs that activate their immune system against the tumor. Scientists feared that vaccination against COVID-19 could reduce the success of cancer treatment or cause severe side effects—until now. A recent study now gives…

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Why interoperability is now an essential part of digital health – and how to prepare for an interoperable future

11/28/2022

Excerpt from the Press Release: Interoperability is a capability that’s been discussed for some time but, until now, a lack of precision in the way the term is used has clouded what it actually means and what it can enable for companies in the healthcare ecosystem. This is changing rapidly, however. Developments both in technology…

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