FRDA

Rgenta Therapeutics to Present at the 19th Annual Huntington's Disease Therapeutics Conference

Retrieved on: 
Wednesday, February 21, 2024
Traffic barrier, MLH1, PMS2, Huntington's disease, GWAS, FXS, Conference, DM1, MSH3, PMS1, RNA, Therapy, FAN1, Gene, FRDA, CSO, Fragile X syndrome, Friedreich's ataxia, DNA, Vaccine

CAMBRIDGE, Mass., Feb. 21, 2024 /PRNewswire/ -- Rgenta Therapeutics ("Rgenta" or the "Company") today announced it will participate at the 19th Annual Huntington's Disease Therapeutics Conference being held February 26-29, 2024, in Palm Springs, California.

Key Points: 
  • CAMBRIDGE, Mass., Feb. 21, 2024 /PRNewswire/ -- Rgenta Therapeutics ("Rgenta" or the "Company") today announced it will participate at the 19th Annual Huntington's Disease Therapeutics Conference being held February 26-29, 2024, in Palm Springs, California.
  • As part of the annual conference, Travis Wager, Ph.D., President & CSO of Rgenta will participate in the Targeting the DNA repair machinery to modulate somatic instability session on Tuesday, February 27, 2024 at 2:00 PM PT.
  • To date, there has been a lack of successful strategies for directly targeting the PMS1 protein with small molecule inhibitors.
  • For more information about the event, please visit the conference website .

Prime Medicine Reports Fourth Quarter and Full Year 2022 Financial Results and Provides Business Update

Retrieved on: 
Thursday, March 9, 2023
LNP, GAA, FXN, CMC, AAV, Chemistry, R, CF, Quarter, Stem cell, Administration, Patient, Chronic granulomatous disease, Mitochondrion, Eye, HSC, CFTR, Research and development, Research, Central nervous system, Liver, Swelling, Gene expression, Investment, Cell, WD, Cystic fibrosis, Cystic fibrosis transmembrane conductance regulator, FRDA, CGD, Pharmaceutical industry, Vaccine, Video game, Cryptocurrency, IND

CAMBRIDGE, Mass., March 09, 2023 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, today reported financial results and provided a business update for the fourth quarter and full year ended December 31, 2022.

Key Points: 
  • “In 2022, we made important progress in our efforts to build Prime Medicine and demonstrate the promise of Prime Editors as a potentially best-in-class genetic medicine approach,” said Keith Gottesdiener, M.D., President and Chief Executive Officer of Prime Medicine.
  • Prime Medicine expects the following activities and next steps to drive the Prime Editing platform forward:
    Initiate investigational new drug (IND)-enabling studies of PM359 for the treatment of CGD in 2023.
  • Maximize Prime Editing’s broad therapeutic potential and create value through strategic business development that extends the reach and impact of Prime Editing to areas beyond Prime Medicine’s current areas of focus.
  • Fourth Quarter and Full Year 2022 Financial Results:
    Research and Development (R&D) Expenses: R&D expenses were $29.1 million for the fourth quarter of 2022 and $86.7 million for the year ended December 31, 2022, as compared to $52.9 million for the fourth quarter of 2021 and $70.6 million for the year ended December 31, 2021.

Prime Medicine Announces Recent Progress and Highlights 2023 Strategic Priorities

Retrieved on: 
Monday, January 9, 2023
Disease, FRDA, Genome, Cystic fibrosis transmembrane conductance regulator, Swelling, CGD, Safety data sheet, Respiratory tract, GAA, Tissue, Cystic fibrosis, CD34, CMC, AAV, Heart, Failure rate, CFTR, Mitochondrion, Culture, DNA, LNP, Safety, Mutation, IPSC, Gene editing, Central nervous system, Hepatocyte, Patient, Degenerative disease, CF, Bronchiole, Neuron, PCSK9, Ataxia, Liver, Human, FXN, Inflammation, Chronic granulomatous disease, Eye, Investment, Prime editing, Vaccine, Pharmaceutical industry, Medical device, IND, WD, Frataxin

CAMBRIDGE, Mass., Jan. 09, 2023 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, today provided an update on recent advancements of its Prime Editing technology and progress across its initial pipeline of eighteen programs, and outlined its strategic priorities and expected milestones for 2023.

Key Points: 
  • Today, Prime Medicine announced new preclinical data demonstrating that Prime Editing-mediated removal of pathological repeats in vitro results in correction of hypermethylation at the FXN gene, restoring genetic function back to wild-type levels.
  • Off-target Safety Data: Prime Medicine is progressing a comprehensive suite of assays to evaluate the potential off-target activity of its Prime Editors.
  • Prime Medicine is using PCSK9 as a model system for developing its modular LNP delivery platform to the liver.
  • Prime Medicine expects the following activities and next steps to drive the Prime Editing platform forward:
    Nominate first development candidate for CGD in 1Q 2023.