Homologous recombination

Homology Medicines Announces First Data from IND-Enabling Studies with GTx-mAb Candidate HMI-104 for PNH, Which Demonstrated Sustained Expression of Functional C5mAb Levels, at the ASGCT Annual Meeting

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Tuesday, May 16, 2023
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BEDFORD, Mass., May 16, 2023 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today six presentations supporting its gene editing, gene therapy and GTx-mAb programs at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting, including the first data from IND-enabling studies with HMI-104, the anti-C5 GTx-mAb development candidate for paroxysmal nocturnal hemoglobinuria (PNH). These preclinical data demonstrated that a one-time administration of HMI-104 resulted in sustained expression of C5 monoclonal antibody (C5mAb) levels, supporting the use of a one-time vectorized approach for PNH to enable continuous antibody production.

Key Points: 
  • These preclinical data demonstrated that a one-time administration of HMI-104 resulted in sustained expression of C5 monoclonal antibody (C5mAb) levels, supporting the use of a one-time vectorized approach for PNH to enable continuous antibody production.
  • Additionally, Homology highlighted methods to identify genomic sites with improved homologous-recombination (HR)-based gene editing integration, which could be used to enhance and streamline development of future product candidates.
  • These data support the immunosuppression regimen used in Homology’s ongoing gene editing and gene therapy clinical trials.
  • In the poster, “Gene Therapy Candidate for Metachromatic Leukodystrophy (MLD): Optimization of HMI-202 Leading to HMI-204 Nomination,” Homology highlighted preclinical data on its optimized HMI-204 gene therapy candidate for MLD.

Allarity Therapeutics Doses First Patient in Phase 1b Clinical Trial Evaluating Dovitinib and Stenoparib Combination in Advanced Solid Tumors

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Monday, March 20, 2023
DNA, Index, MOA, Therapy, Neoplasm, Poly (ADP-ribose) polymerase, Nutrient, Coronavirus Scientific Advisory Board, Gynecologic Oncology (journal), Moa, Associate, MS, Blood, Downregulation and upregulation, Patient, Mass, Clinical research, HR, MTD, Growth, Fibroblast growth factor receptor, Principal, Ovarian cancer, Death, PARP, PTK, Homologous recombination, Safety, Pharmaceutical industry, Medical imaging, MD

“Having investigated novel combinations of anticancer agents, including a PARP inhibitor and an anti-angiogenic therapeutic, we have seen improved efficacy.

Key Points: 
  • “Having investigated novel combinations of anticancer agents, including a PARP inhibitor and an anti-angiogenic therapeutic, we have seen improved efficacy.
  • The Phase 1b trial is designed with a target enrollment of up to 36 patients with advanced solid tumors, focusing on specific tumor types that Allarity anticipates will be most responsive to the drug combination.
  • Researchers will analyze patient tumor samples retrospectively using Allarity’s DRP® companion diagnostics for stenoparib and dovitinib.
  • Allarity holds exclusive, global commercial rights to both dovitinib and stenoparib.

Aprea Therapeutics Announces Phase II SBIR Award from the National Cancer Institute (NCI)

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Wednesday, February 15, 2023
DNA, Biomarker, Coronavirus Scientific Advisory Board, Homologous recombination, Cancer, Woman, APRE, Laboratory, NCI, Perelman School of Medicine at the University of Pennsylvania, CCNE1, Therapy, ATR, Obstetrics, National Cancer Institute, Bangladesh Technical Education Board, Aprea-Cuccaro clan, Patient, BRCA2, Clinical trial, University, DDR, Associate, Gene, Survival, Disease, ATRN, Growth, Research, WEE1, Gynaecology, DRS, Death, Pharmaceutical industry

Approximately 50% of HGSOCs harbor genetic mutations in BRCA1, BRCA2, or other genes that cause defects in homologous recombination (HR).

Key Points: 
  • Approximately 50% of HGSOCs harbor genetic mutations in BRCA1, BRCA2, or other genes that cause defects in homologous recombination (HR).
  • In addition, Aprea Therapeutics has also developed a highly potent and selective WEE1 inhibitor, ATRN-W1051, with a differentiated structure and potentially preferable pharmacokinetic properties.
  • Dr. Simpkins is a member of the Aprea Therapeutics Scientific Advisory Board and affiliated with the University of Pennsylvania.
  • Research content supported by the National Cancer Institute of the National Institutes of Health under Award Number R44CA278078 is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

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

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Wednesday, January 4, 2023
Immunosuppression, Clinical trial, Homologous recombination, Steroid, RNA transfection, Gene editing, NAB, AAV, MPS, MLD, Hunter syndrome, PKU, PNH, Genome, MPS II, IND, JumpStart, Paroxysmal nocturnal hemoglobinuria, PAH, Phenylketonuria, Pharmaceutical industry, Vaccine

BEDFORD, Mass., Jan. 04, 2023 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today enrollment and site status updates from the pheEDIT Phase 1 gene editing trial with HMI-103 for phenylketonuria (PKU) and the juMPStart Phase 1 gene therapy trial with HMI-203 for Hunter syndrome (MPS II).

Key Points: 
  • Homology expects to provide initial data from the trial mid-year 2023.
  • There are nine active clinical trial sites with more expected to be initiated throughout 2023.
  • The juMPStart trial has five clinical sites in the U.S. and Canada with more expected to be initiated, and initial data is expected in the second half of 2023.
  • Importantly, we plan to share initial clinical data from both trials in 2023, including the first data from our nuclease-free gene editing technology in humans.”
    Homology shared new preclinical data today supporting the immunosuppression regimen incorporated in both the pheEDIT and juMPStart trials.

KSQ Therapeutics Announces First Patient Dosed in Combination Portion of the Ongoing Phase 1 Study of KSQ-4279, a First-In-Class USP1 Inhibitor, in Patients with Advanced Solid Tumors

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Wednesday, December 7, 2022
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This stage of the clinical study will investigate KSQ-4279 across multiple indications, both in combination with a PARP inhibitor and in combination with chemotherapy.

Key Points: 
  • This stage of the clinical study will investigate KSQ-4279 across multiple indications, both in combination with a PARP inhibitor and in combination with chemotherapy.
  • "The initiation of the combination therapy portion of the study is a significant milestone for KSQ.
  • Study KSQ-4279-1101 is a Phase 1 clinical trial expected to enroll approximately 140 patients with advanced solid tumors.
  • KSQ-4279 is currently being evaluated in a Phase 1 study in patients with advanced solid tumors.

Homology Medicines Presents on Design of pheEDIT Trial Evaluating One-Time Nuclease-Free Gene Editing Candidate HMI-103 for PKU at American Society of Human Genetics Meeting

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Thursday, October 27, 2022
Molecule, Therapy, SEC, Company, Phenylketonuria, Security (finance), Antibody, U.S. Securities and Exchange Commission, Society, Private Securities Litigation Reform Act, PAH, Safety, Nasdaq, Paroxysmal nocturnal hemoglobinuria, Disease, Hunter syndrome, PKU, PHE, Human genetics, Patient, American Society of Human Genetics, Homologous recombination, Causality, ASHG, MLD, COVID-19, Gene editing, Capsid, Use, GLOBE, Efficacy, AAV, PNH, Failure, Genome, Vaccine, Pharmaceutical industry, Adult

BEDFORD, Mass., Oct. 27, 2022 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today a presentation on the design of pheEDIT, a Phase 1, open-label, dose-escalation study evaluating one-time gene editing candidate HMI-103 in adults with phenylketonuria (PKU). The presentation included preclinical data demonstrating efficacy in a PKU murine model and precision of editing in a humanized murine model. During the American Society of Human Genetics (ASHG) Annual Meeting, the Company also presented data that demonstrated single-molecule, modified base sequencing can aid in the characterization, design and optimization of AAV vectors.

Key Points: 
  • The presentation included preclinical data demonstrating efficacy in a PKU murine model and precision of editing in a humanized murine model.
  • During the American Society of Human Genetics (ASHG) Annual Meeting, the Company also presented data that demonstrated single-molecule, modified base sequencing can aid in the characterization, design and optimization of AAV vectors.
  • The programs preclinical data showed long-term Phe normalization in the PKU model following a single I.V.
  • The Companys clinical programs include HMI-103, a gene editing candidate for phenylketonuria (PKU); HMI-203, an investigational gene therapy for Hunter syndrome; and HMI-102, an investigational gene therapy for adults with phenylketonuria (PKU).

Antengene Announces HREC Approval in Australia for the Phase I Trial of the Small Molecule ATR Inhibitor ATG-018

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Tuesday, June 7, 2022
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SHANGHAI and HONG KONG, June 6, 2022 /PRNewswire/ -- Antengene Corporation Limited ("Antengene" SEHK: 6996.HK), a leading commercial-stage global biopharmaceutical company dedicated to discovering, developing and commercializing first-in-class and/or best-in-class therapeutics in hematology and oncology, today announced that Antengene has received approval by the Bellberry Human Research Ethics Committee (HREC) in Australia to initiate the Phase I Trial of ATG-018 in patients with advanced solid tumors and hematologic malignancies (ATRIUM Trial).

Key Points: 
  • ATG-018 has the potential asthe first orally-available,small molecule ATR kinase inhibitor entering clinical development in Australia.
  • ATG-018 is an orally-available, potent, selective small molecule ATR inhibitor.
  • Icon Brisbane in Australia is the lead site for the study, which will be conducted at five sites across Australia.
  • Discovered by the internal R&D Team at Antengene, ATG-018 is an oral, potent, selective small molecule inhibitor targeting ataxia telangiectasia and Rad3-associated (ATR) kinase.

Black Diamond Therapeutics Appoints Sergey Yurasov, M.D., Ph.D., as Chief Medical Officer

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Wednesday, June 1, 2022
Diamond, Leadership, Achievement, Large-cell lung carcinoma, Company, Rucaparib, Program, Board of directors, EGFR, Glioblastoma, M26 Modular Accessory Shotgun System, First Pavlov State Medical University of St. Petersburg, Immunity (medical), Roche, Gaps, Cancer, ImClone Systems, Senior, GBM, Investigational New Drug, Research institute, Nasdaq, Ovarian cancer, Growth, Eli Lilly and Company, Â, Patient, GLOBE, Private Securities Litigation Reform Act, MAP, NEW, Clovis Oncology, Board, Drug discovery, Merck, NSCLC, Homologous recombination, U.S. Securities and Exchange Commission, Hematology, Black Diamond, Pharmaceutical industry, Risk management, IND

CAMBRIDGE, Mass. and NEW YORK, June 01, 2022 (GLOBE NEWSWIRE) -- Black Diamond Therapeutics, Inc. (Nasdaq: BDTX), a precision oncology medicine company pioneering the discovery and development of MasterKey therapies, today announced the appointment of Sergey Yurasov, M.D., Ph.D., as Chief Medical Officer. In conjunction with the appointment of Dr. Yurasov, Karsten Witt, M.D., is transitioning from his current role as the Company’s Interim Chief Medical Officer to a clinical advisory role.

Key Points: 
  • and NEW YORK, June 01, 2022 (GLOBE NEWSWIRE) -- Black Diamond Therapeutics, Inc. (Nasdaq: BDTX), a precision oncology medicine company pioneering the discovery and development of MasterKey therapies, today announced the appointment of Sergey Yurasov, M.D., Ph.D., as Chief Medical Officer.
  • We are delighted to welcome Sergey to Black Diamond as Chief Medical Officer.
  • Dr. Yurasov joins Black Diamond Therapeutics from Nuvation Bio, an oncology therapeutics company.
  • Dr. Yurasov succeeds Dr. Witt, who was Black Diamonds founding Chief Medical Officer and will continue to support the Company in an advisory capacity.

Clinical Development Update from Portfolio Company LogicBio

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Monday, May 9, 2022
Capsid, Generides, Homologous recombination, IND, Nasdaq, SUNRISE, EMA, Mouse, European Medicines Agency, Biotechnology, Genome, AAV, Food, Methylmalonic acidemia, Safety, Time, Patient, Review, Survival, Methylmalonyl-CoA, FDA, Letter, Hepatocyte, Cancer, Industry, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, DNA, Lifting, Tissue, Thrombotic microangiopathy, Methylmalonyl-CoA mutase, List of life sciences, Liver, TMA, Time-invariant system, Growth, Risk, Therapy, Pharmaceutical industry, Vaccine, MMA

As a listed company, we are able to bring this exciting growth phase of our industry to a broader range of investors.

Key Points: 
  • As a listed company, we are able to bring this exciting growth phase of our industry to a broader range of investors.
  • In its letter, the FDA acknowledged that the company satisfactorily addressed all clinical hold issues.
  • In connection with the lifting of the clinical hold, LogicBio amended the SUNRISE protocol in a manner that reflected its dialogue with the FDA.
  • LogicBio Therapeutics is a clinical-stage genetic medicine company pioneering genome editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood.

InvestmentPitch Media Video Discusses Rakovina Therapeutics and its Presentation of Preclinical Data Supporting Potential Broad Anticancer Activity of kt-4000 Series at the American Association of Cancer Research Annual General Meeting

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Thursday, April 14, 2022
C1-inhibitor, News, Awareness, Medicine, DNA, Annual general meeting, Association, Combination, Research, Induced cell cycle arrest, Homologous recombination, GLOBE, CFO, Death, Patient, Cell, Bifunctional, Survivor, PARP, Suggestion, Immune system, BRCA, Therapy, Health Canada, DNA repair, American Association, FDA, Cancer, Risk, IR, AACR, Survival, Pharmaceutical industry, Vaccine

The Annual Meeting highlights the work of the best minds in cancer research from institutions worldwide.

Key Points: 
  • The Annual Meeting highlights the work of the best minds in cancer research from institutions worldwide.
  • Rakovina Therapeutics was established in 2020 to develop new cancer treatments based on novel DNA-damage response technologies.
  • Rakovina Therapeutics has paid a fee not exceeding $2,000 in cash to have its current news release produced in video format.
  • The company specializes in producing short videos based on significant news releases, research reports and other content of interest to investors.