Month: August 2022

Disc Medicine Initiates BEACON, a Phase 2 Clinical Study of Bitopertin in Patients with Erythropoietic Protoporphyria (EPP) and X-linked Protoporphyria (XLP)

08/22/2022

BEACON study will evaluate bitopertin as a potential disease-modifying treatment for patients with Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP) Key assessments include changes in protoporphyrin IX levels, safety, tolerability and measures of photosensitivity; preliminary data expected by 1H 2023 Excerpt from the Press Release: WATERTOWN, Mass., Aug. 10, 2022 /PRNewswire/ — Disc Medicine, a clinical-stage biotechnology…

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Cure Rare Disease Receives FDA Approval to Administer First-in-Human CRISPR Therapeutic

08/22/2022

The first-of-its-kind, CRISPR-based therapeutic aims to stop the progression of Duchenne muscular dystrophy in a single-patient dosing Excerpt from the Press Release: BOSTON–(BUSINESS WIRE)–Cure Rare Disease (CRD) – a Boston-based 501c3 nonprofit biotech – announces the approval from the U.S. Food and Drug Administration (FDA) to administer its very first therapeutic. The drug, named CRD-TMH-001, treats…

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BostonGene Announces Publication in Cancer Cell Revealing the Development of a Machine Learning Cellular Deconvolution Algorithm that Accurately Reconstructs the Tumor Microenvironment and Supports Oncology Clinical Decision-making

08/19/2022

Excerpt from the Press Release: WALTHAM, Mass.–(BUSINESS WIRE)–BostonGene today announced the online publication of the manuscript, “Precise reconstruction of the tumor microenvironment using bulk RNA-seq and a unique machine learning algorithm trained on artificial transcriptomes”in Cancer Cell, a premier peer-reviewed scientific journal that publishes high impact results in cancer research and oncology.The study demonstrated the ability…

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Olema Oncology Advances OP-1250 into Phase 2 Monotherapy Expansion in Patients with ER+/HER2- Advanced Breast Cancer

08/19/2022

Excerpt from the Press Release: SAN FRANCISCO, Aug. 09, 2022 (GLOBE NEWSWIRE) — Olema Pharmaceuticals, Inc. (“Olema” or “Olema Oncology,” Nasdaq: OLMA), today announced the advancement of OP-1250, a complete estrogen receptor (ER) antagonist (CERAN) and a selective ER degrader (SERD), into Phase 2 clinical development for the treatment of ER+/HER2- metastatic breast cancer. “We are excited…

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Biora Therapeutics Announces Successful Completion of Device Performance Study in Ulcerative Colitis Patients for its Targeted Therapeutics Platform

08/19/2022

All Devices Successfully Detected Colon Entry in Patients with Active Ulcerative Colitis Excerpt from the Press Release: SAN DIEGO, Aug. 10, 2022 (GLOBE NEWSWIRE) — Biora Therapeutics, Inc. (Nasdaq: BIOR), the biotech company that is reimagining therapeutics, today announced topline results from its recently completed study PM-602: A Scintigraphic Study to Evaluate the Localization and Delivery…

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Nabriva Therapeutics Completes Patient Enrollment in Phase 1 Trial of XENLETA® (lefamulin) in Adult Patients with Cystic Fibrosis

08/18/2022

– Topline Data Expected First Quarter 2023 – Excerpt from the Press Release: DUBLIN, Ireland and FORT WASHINGTON, Pa., Aug. 11, 2022 (GLOBE NEWSWIRE) — Nabriva Therapeutics plc (NASDAQ: NBRV), a biopharmaceutical company engaged in the commercialization and development of innovative anti-infective agents to treat serious infections, announced the completion of patient enrollment in its…

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iNtRON developed New CRISPR/Cas technology specific for Bacteriophage as PHAGERUS® Platform

08/18/2022

CRISPR/Cas system for bacteria that are not E. coli or Streptococcus pyogenes Customized gene editing technology for unique modified-bacteriophages To validate efficacy of SARS-CoV-2 mimotopes loaded PHAGERUS® platform Excerpt from the Press Release: BOSTON and SEOUL, South Korea, Aug. 9, 2022 /PRNewswire/ — iNtRON Biotechnology (“iNtRON”, www.intodeworld.com) announced today that iNtRON has developed its unique CRISPR/Cas system customized for the genetic modification of certain bacteria…

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Deciphera Pharmaceuticals, Inc. Announces Publication of INTRIGUE Phase 3 Clinical Study Results in Journal of Clinical Oncology

08/18/2022

– Efficacy Observed with QINLOCK® was Comparable to Sunitinib with a More Favorable Safety and Tolerability Profile in GIST Patients Previously Treated with Imatinib – Excerpt from the Press Release: WALTHAM, Mass.–(BUSINESS WIRE)–Deciphera Pharmaceuticals, Inc. (NASDAQ: DCPH), a biopharmaceutical company focused on discovering, developing, and commercializing important new medicines to improve the lives of people with cancer,…

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Lucira Announces Health Canada Authorization of First and Only 99% Accurate At-Home Covid & Flu Test

08/17/2022

Excerpt from the Press Release: EMERYVILLE, Calif., Aug. 11, 2022 (GLOBE NEWSWIRE) — Lucira Health, Inc. (Nasdaq: LHDX) (“Lucira Health,” “Lucira” or the “Company”), a medical technology company focused on the development and commercialization of transformative and innovative infectious disease tests, today announced that Health Canada has granted Authorization under Interim Order for the emergency…

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Graphite Bio Doses First Patient with Investigational Gene Editing Therapy GPH101 for Sickle Cell Disease

08/17/2022

GPH101, now called nulabeglogene autogedtemcel (nula-cel), designed to directly correct the genetic mutation that causes sickle cell disease Initial proof-of-concept data from Phase 1/2 CEDAR trial anticipated in mid-2023 Excerpt from the Press Release: SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Graphite Bio, Inc. (Nasdaq: GRPH), a clinical-stage, next-generation gene editing company harnessing the power of high-efficiency precision…

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