Friedreich's ataxia

Center for Breakthrough Medicines Launches Genesis Vector Manufacturing Solutions™ with New State-of-the-Art Vector Suites to Accelerate the Commercialization of Advanced Therapies

Retrieved on: 
Thursday, December 8, 2022
CDMO, Partnership, Patient, DNA, CBM, Science, GMP, Integration, Disease, Friedreich's ataxia, Sterilization, COGS, Multimedia, Documentation, CGMP, Vaccine, Pharmaceutical industry, Fine chemical

KING OF PRUSSIA, Pa., Dec. 8, 2022 /PRNewswire/ -- The Center for Breakthrough Medicines (CBM), a contract development, manufacturing, and testing organization (CDMO) has launched Genesis Vector Manufacturing Solutions™ to help innovator companies reimagine vector manufacturing by simplifying the value chain and accelerate their path to clinic and commercial launch.

Key Points: 
  • CBM's Genesis Vector Manufacturing Solutions is an end-to-end offering that includes plasmid manufacturing, first-time right process development and high-throughput GMP vector manufacturing suites to accelerate development and manufacturing timelines.
  • Integrated testing & analytics, supply chain, and regulatory services co-located with manufacturing at a single site drastically simplifies the vector value chain.
  • Each GMP vector manufacturing suite supports batch output 2-5 times greater than traditional designs while maintaining maximum flexibility in production platforms.
  • All suites are equipped with real-time process monitoring through digital networks and livestreams of the operations for truly transparent manufacturing.

Wren Taps Vertex Veteran Executives and Other Leaders to Advance First-in-Class Small-Molecule Drugs into Clinical Testing for Neurodegenerative Diseases

Retrieved on: 
Thursday, December 8, 2022
Novartis Institute for Tropical Diseases, Therapy, Degenerative disease, Friedreich's ataxia, Neurodegeneration, Thomson, Biomedical Research, Agios Pharmaceuticals, NDA, Alzheimer's disease, Sickle cell disease, Doctor of Philosophy, Drug discovery, Biophysics, Research, Hepatitis, Science, Ideation, IEEE Corporate Innovation Recognition, Schering-Plough, Autism, Vertex, Huntington's disease, FDA, Drug development, Pharmaceutical Development and Technology, ALS, NIBR, Endocrinology, Vertex Pharmaceuticals, Human, Translation, Senior, Connelly, Head, Hepatitis C, Inflammation, Parkinson's disease, Decibel, Cystic fibrosis, Ipsen, Fine chemical, Pharmaceutical industry, Management, Chemistry, Wren, Ivosidenib, EU, Sanofi, DRS

BOSTON, Dec. 8, 2022 /PRNewswire/ -- Wren Therapeutics announced today a number of senior leadership appointments as the company advances its first investigational therapies toward the clinic, based on the company's breakthrough approach to discovering small molecules that precisely target and shut down the sources of oligomer and aggregate generation in neurodegenerative diseases.

Key Points: 
  • The company recently welcomed Vertex Pharmaceuticals veterans John Thomson, PhD and Patrick Connelly, PhD as its Chief Scientific Officer and first Chief Innovation Officer, respectively.
  • Dr. Thomson and Dr. Connelly are Vertex founding scientists who worked together on leadership teams that discovered and developed Vertex's seven FDA-approved drugs.
  • "We couldn't be more pleased to welcome John, Pat, Mike, Janeta, and Rajeev to Wren's teamall proven executives in their fields.
  • With this lineup of experienced talent, Wren has the leadership team to break open the field of neurodegenerative diseases targeting the molecular mechanisms at the source of toxic oligomer generation."

Larimar Therapeutics to Present at the 34th Annual Piper Sandler Healthcare Conference

Retrieved on: 
Monday, November 21, 2022
Therapy, Friedreich's ataxia, Conference, Pharmaceutical industry, Vaccine

BALA CYNWYD, Pa., Nov. 21, 2022 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases,today announced that company management will present and participate in 1x1 investor meetings at the 34th Annual Piper Sandler Healthcare Conference, which is taking place in New York, NY from November 29 December 1, 2022.

Key Points: 
  • BALA CYNWYD, Pa., Nov. 21, 2022 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases,today announced that company management will present and participate in 1x1 investor meetings at the 34th Annual Piper Sandler Healthcare Conference, which is taking place in New York, NY from November 29 December 1, 2022.
  • The Companys presentation will take place on Thursday, December 1, 2022, at 10:10 AM ET.
  • Larimar Therapeutics, Inc. (Nasdaq: LRMR), is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases.
  • Larimar also plans to use its intracellular delivery platform to design other fusion proteins to target additional rare diseases characterized by deficiencies in intracellular bioactive compounds.

Larimar Therapeutics Reports Third Quarter 2022 Operating and Financial Results

Retrieved on: 
Thursday, November 10, 2022
GLOBE, Review, Marketing, U.S. Securities and Exchange Commission, FA, Food, Nasdaq, Research, Annual report, Technology, Friedreich's ataxia, LRMR, Patent, COVID-19, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Therapy, SEC, Company, Form 10-K, Clinical trial, Terminology, Patient, 1836 U.S. Patent Office fire, Security (finance), Ataxia, FDA, Pharmaceutical industry, Reinsurance, Larimar, MD

Cash of $124.7 million at September 30, 2022 provides projected cash runway into 2H 2024

Key Points: 
  • Cash of $124.7 million at September 30, 2022 provides projected cash runway into 2H 2024
    BALA CYNWYD, Pa., Nov. 10, 2022 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases,today reported its third quarter 2022 operating and financial results.
  • In September 2022, Larimar raised net proceeds of approximately $75.2 million through an underwritten offering of common stock.
  • Research and development expenses for the third quarter of 2022 were $5.6 million compared to $14.0 million for the third quarter of 2021.
  • General and administrative expenses for the third quarter of 2022 were $2.9 million compared to $2.7 million for the third quarter of 2021.

Larimar Therapeutics to Participate in the Guggenheim 4th Annual Immunology & Neurology Day

Retrieved on: 
Monday, November 7, 2022
Nasdaq, Therapy, Friedreich's ataxia, Vaccine

Larimar Therapeutics, Inc. (Nasdaq: LRMR), is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases.

Key Points: 
  • Larimar Therapeutics, Inc. (Nasdaq: LRMR), is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases.
  • Larimars lead compound, CTI-1601, is being developed as a potential treatment for Friedreich's ataxia.
  • Larimar also plans to use its intracellular delivery platform to design other fusion proteins to target additional rare diseases characterized by deficiencies in intracellular bioactive compounds.
  • For more information, please visit: https://larimartx.com .

Design Therapeutics Highlights Pipeline Progress and Upcoming Milestones and Reports Third Quarter 2022 Financial Results

Retrieved on: 
Thursday, November 3, 2022
G&A, Private Securities Litigation Reform Act, Science, Therapy, International Congress on Tuberculosis, MAD, Industry, GLOBE, Severe cognitive impairment, Degenerative disease, D, Adult, Safety, IND, Pharmacokinetics, Mutation, FA, Nasdaq, Patient, Gene expression, Nature, Program, Friedreich's ataxia, U.S. Securities and Exchange Commission, Clinical trial, ICAR, GAA, FXN, Risk, R, Fuchs, Disease, Security (finance), SEC, SAD, Treatment, Drug, DM1, Pharmaceutical industry, Vaccine, Design, Research

CARLSBAD, Calif., Nov. 03, 2022 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today highlighted recent progress and anticipated upcoming milestones across its clinical and research-stage pipeline of novel GeneTAC™ small molecules and reported third quarter 2022 financial results.

Key Points: 
  • In addition, we recently initiated dosing in the multiple-ascending dose trial, keeping us on-track to complete Phase 1 mid next year.
  • With a solid cash position, expert team and strong science behind us, Im confident in our ability to execute our milestones.
  • Design Therapeutics is a clinical-stage biotechnology company developing a new class of therapies based on its platform of GeneTAC gene targeted chimera small molecules.
  • You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof.

Larimar Therapeutics Announces Issuance of U.S. Patent Providing Composition of Matter Protection for CTI-1601

Retrieved on: 
Thursday, October 20, 2022
European Medicines Agency, Union, Method, SEC, Therapy, Royal commission, LRMR, Private Securities Litigation Reform Act, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Patient, Annual report, Friedreich's ataxia, Nasdaq, Orphan drug, Form 10-K, Food, Company, Food and Drug Administration, Mitochondrion, Patent, Indiana University, GLOBE, Intellectual property, FDA, European Commission, Pharmaceutical industry, Larimar, MD

BALA CYNWYD, Pa., Oct. 20, 2022 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases,today announced the issuance of U.S. Patent No.

Key Points: 
  • BALA CYNWYD, Pa., Oct. 20, 2022 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases,today announced the issuance of U.S. Patent No.
  • The patent, titled, Materials and Methods for Treating Friedreich's Ataxia, provides composition of matter protection for CTI-1601 into at least July 2040.
  • Larimar has an exclusive license to the patent per a prior agreement with Indiana University.
  • This new patent represents a foundational component of our intellectual property portfolio, as it extends our patent protection for CTI-1601 by more than fourteen years, said Carole Ben-Maimon, MD, President and Chief Executive Officer of Larimar.

Larimar Therapeutics Announces Oral and Poster Presentations at the Upcoming International Congress for Ataxia Research

Retrieved on: 
Wednesday, October 19, 2022
GLOBE, Frataxin, International Congress on Tuberculosis, Therapy, Safety, Pharmacokinetics, Ataxia, Friedreich's ataxia, Research, Nasdaq, Pharmaceutical industry

BALA CYNWYD, Pa., Oct. 19, 2022 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases,today announced that data from the Companys Phase 1 clinical program evaluating CTI-1601 as a potential treatment for Friedreich's ataxia will be featured in three presentations at the upcoming International Congress for Ataxia Research .

Key Points: 
  • BALA CYNWYD, Pa., Oct. 19, 2022 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases,today announced that data from the Companys Phase 1 clinical program evaluating CTI-1601 as a potential treatment for Friedreich's ataxia will be featured in three presentations at the upcoming International Congress for Ataxia Research .
  • The conference will take place November 1 4, 2022, in Dallas, Texas.
  • Details on the presentations are shown below.
  • Safety and Pharmacokinetics of Single and 13 Day Multiple-Dose Administration of CTI-1601, a Frataxin Replacement Therapy for Friedreichs Ataxia

Design Therapeutics to Present Preclinical Data Highlighting the Potential of its GeneTAC™ Small Molecule, DT-216, for the Treatment of Friedreich Ataxia at ICAR 2022

Retrieved on: 
Monday, October 17, 2022
Gene expression, FA, U.S. Securities and Exchange Commission, Cardiomyopathy, Arrhythmia, Disease, Therapy, SEC, Risk, IND, Primate, FXN, Patient, ICAR, GAA, RATS, International Congress on Tuberculosis, Research, Program, Investigational New Drug, Security (finance), Nasdaq, Private Securities Litigation Reform Act, Neuron, Mutation, Industry, Pharmacokinetics, Nature, Adult, Diabetes, Safety, CNS, GLOBE, Degenerative disease, Clinical trial, Heart, Friedreich's ataxia, Pharmaceutical industry, Vaccine, Nikolaus Friedreich, Design

The data were included in the companys Investigational New Drug (IND) application for DT-216, which is currently being evaluated in a Phase 1 clinical trial.

Key Points: 
  • The data were included in the companys Investigational New Drug (IND) application for DT-216, which is currently being evaluated in a Phase 1 clinical trial.
  • DT-216 is a GeneTAC small molecule designed to specifically target the GAA repeat expansion mutation and restore endogenous FXN transcription.
  • The preclinical data support the potential for DT-216 to restore FXN gene expression, improve mitochondrial function and address the root cause of FA.
  • You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof.

Design Therapeutics to Present at the 2022 Jefferies Cell and Genetic Medicine Summit

Retrieved on: 
Thursday, September 22, 2022
Friedreich's ataxia, GLOBE, Degenerative disease, Nasdaq, Disease, Therapy, Program, Pharmaceutical industry

CARLSBAD, Calif., Sept. 22, 2022 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today reported that Joo Siffert, M.D., president and chief executive officer, and Sean Jeffries, Ph.D., chief operating officer, will participate in a fireside chat during the 2022 Jefferies Cell and Genetic Medicine Summit on Thursday, September 29, 2022, at 10:00 a.m.

Key Points: 
  • CARLSBAD, Calif., Sept. 22, 2022 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today reported that Joo Siffert, M.D., president and chief executive officer, and Sean Jeffries, Ph.D., chief operating officer, will participate in a fireside chat during the 2022 Jefferies Cell and Genetic Medicine Summit on Thursday, September 29, 2022, at 10:00 a.m.
  • A live webcast will be available in the investor section of the company's website at www.designtx.com and will be archived for 30 days following the presentation.
  • Design Therapeutics is a clinical-stage biotechnology company developing a new class of therapies based on its platform of GeneTAC gene targeted chimera small molecules.
  • The companys GeneTAC molecules are designed to either dial up or dial down the expression of a specific disease-causing gene to address the underlying cause of disease.