Frataxin

Larimar Therapeutics Reports Third Quarter 2023 Operating and Financial Results

Retrieved on: 
Tuesday, November 14, 2023
Research, Safety, Therapy, IP, INN, International, Frataxin, Friedreich's ataxia, Clinical trial, Insurance, Patient, OLE, FDA, FA, Pharmacokinetics, Gene expression, Caregiver, Ataxia, CMO, USAN, Skin, Horizon, Amgen, Pharmaceutical industry, Cryptocurrency, Larimar, MD

“For the OLE trial, initiation remains on track for the first quarter of 2024, and we expect to report interim data later that year in the fourth quarter.

Key Points: 
  • “For the OLE trial, initiation remains on track for the first quarter of 2024, and we expect to report interim data later that year in the fourth quarter.
  • Research and development expenses for the third quarter of 2023 were $6.6 million compared to $5.6 million for the third quarter of 2022.
  • General and administrative expenses for the third quarter of 2023 were $3.8 million compared to $2.9 million for the third quarter of 2022.
  • Other income (expense), net was $1.3 million of income in the third quarter of 2023 compared to $0.2 million in the third quarter of 2022.

Vesigen Therapeutics Awarded Grant from Friedreich’s Ataxia Research Alliance (FARA) to Develop a Targeted Genome Editing Therapeutic Strategy

Retrieved on: 
Tuesday, March 14, 2023
Biotechnology, Health, Genetics, Science, Research, Tissue, Friedreich's ataxia, FARA, DSM-IV codes, Doctor of Philosophy, Neuron, CRISPR, Neurodegenerative disease, Genome, Genetic disorder, Seminal vesicles, Patient, Multimedia, Engineering, Therapy, Drug development, ARMM (Usenet), FA, Vaccine, Pharmaceutical industry, Frataxin

Vesigen will evaluate its proprietary technology to deliver CRISPR-Cas genome editing complexes as a non-viral disease-modifying strategy for patients diagnosed with the neurodegenerative disease Friedreich's Ataxia (FA).

Key Points: 
  • Vesigen will evaluate its proprietary technology to deliver CRISPR-Cas genome editing complexes as a non-viral disease-modifying strategy for patients diagnosed with the neurodegenerative disease Friedreich's Ataxia (FA).
  • The FARA Grant Program funds competitive grants across the spectrum from basic research through drug development and clinical research programs.
  • “The work being done by Vesigen Therapeutics has the potential to be transformative for FA patients,” says Jennifer Farmer, CEO of FARA.
  • “We are grateful for Dr. Nabhan and his team for their work on this targeted genome editing strategy.”
    This is the first grant in neurological disorders received by Vesigen Therapeutics.

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.

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

Larimar Therapeutics Announces Upcoming Poster Presentations at the 4th Pan American Parkinson’s Disease and Movement Disorders Congress

Retrieved on: 
Thursday, May 19, 2022
American, Nasdaq, Therapy, Friedreich's ataxia, Movement disorders, Treatment, Frataxin, Administration, Ataxia, GLOBE, Pharmaceutical industry

BALA CYNWYD, Pa., May 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 two upcoming poster presentations at the 4th Pan American Parkinsons Disease and Movement Disorders Congress, which is taking place May 26 28, 2022 at the Hyatt Regency Miami in Miami, Florida.

Key Points: 
  • BALA CYNWYD, Pa., May 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 two upcoming poster presentations at the 4th Pan American Parkinsons Disease and Movement Disorders Congress, which is taking place May 26 28, 2022 at the Hyatt Regency Miami in Miami, Florida.
  • Details on the poster presentations are shown below.
  • Tissue Frataxin Increases After Administration of CTI-1601, a Frataxin Replacement Therapy in Development for the Treatment of Friedreichs Ataxia

Larimar Therapeutics Reports Fourth Quarter and Full Year 2021 Operating and Financial Results

Retrieved on: 
Friday, March 25, 2022
Company, Therapy, NHP, Frataxin, Form 10-K, Marketing, Clinical trial, European Medicines Agency, Annual report, Skin, Research, SEC, Toxicology, MAD, Ataxia, Medicine, Food, Tissue, Communication, FA, LRMR, Nasdaq, COVID-19, Șaeș, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, GLOBE, EMA, Accounting, Terminology, FDA, Insurance, Friedreich's ataxia, Patient, Pharmaceutical industry, Larimar, MD

BALA CYNWYD, Pa., March 25, 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 full year 2021 operating and financial results.

Key Points: 
  • BALA CYNWYD, Pa., March 25, 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 full year 2021 operating and financial results.
  • In May 2021, Larimar reported positive topline data from its Phase 1 Friedreichs ataxia (FA) program.
  • Research and development expenses for the fourth quarter of 2021 were $6.3 million compared to $10.6 million for the fourth quarter of 2020.
  • General and administrative expenses for the fourth quarter of 2021 were $2.8 million compared to $3.8million for the fourth quarter of 2020.

Larimar Therapeutics Reports Second Quarter 2021 Operating and Financial Results

Retrieved on: 
Thursday, August 12, 2021
Jive, SEC, European Medicines Agency, Nasdaq, Program, SAD, Frataxin, Standby Equity Distribution Agreement, Medicine, GLOBE, Letter, FDA, Șaeș, LRMR, Pharmacology, Marketing, Skin, Clinical trial, Form 10-K, Toxicology, Insurance, Terminology, Knowledge, Tissue, Annual report, FA, Company, Research, Accounting, Investment, Time, EMA, MAD, COVID-19, Therapy, Patient, Friedreich's ataxia, NHP, Cryptocurrency, Security (finance), Pharmaceutical industry

- Cash and investments of $70.6 million as of June 30, 2021

Key Points: 
  • - Cash and investments of $70.6 million as of June 30, 2021
    - Closed $20 million equity financing on July 2, 2021
    BALA CYNWYD, Pa., Aug. 12, 2021 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases,today reported its second quarter and year to date June 30, 2021 operating and financial results.
  • In May 2021, Larimar received European Medicines Agency (EMA) Priority Medicines (PRIME) designation for CTI-1601 in FA.
  • Research and development expenses for the second quarter of 2021 were $9.1 million compared to $8.9 million for the second quarter of 2020.
  • General and administrative expenses for the second quarter of 2021 were $3.4 million, compared to $2.5million for the second quarter of 2020.

Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) Drug Development Research Report 2021 - ResearchAndMarkets.com

Retrieved on: 
Wednesday, July 14, 2021
Genetics, Pharmaceutical, Health, Branches of biology, Frataxin, Proteins, Friedreich's ataxia, Ataxin, Mitochondrion

The "Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) - Drugs In Development, 2021" drug pipelines has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) - Drugs In Development, 2021" drug pipelines has been added to ResearchAndMarkets.com's offering.
  • Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) pipeline Target constitutes close to 21 molecules.
  • Frataxin Mitochondrial - Drugs In Development, 2021, outlays comprehensive information on the Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type.
  • It also reviews key players involved in Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) targeted therapeutics development with respective active and dormant or discontinued projects.

Exicure Announces Upcoming Neuroscience Pipeline Update at Virtual R&D Day

Retrieved on: 
Thursday, July 8, 2021
Science, Other Science, Research, Pharmaceutical, Oncology, Health, Genetics, Other Health, Neurological disorders, Branches of biology, Nervous system, Rare diseases, Lipid storage disorders, Frataxin, Proteins, Batten disease, Friedreich's ataxia, CLN3, Nav1.7, Neurodegeneration, Friedreich’s Ataxia, SCN9A, CLN3 Batten Disease, Exicure, Inc., FRIEDREICH’S ATAXIA, SCN9A, CLN3 BATTEN DISEASE, EXICURE, INC.

The event will showcase Exicures neuroscience pipeline, including its lead program for Friedreichs Ataxia (FA), XCUR-FXN, which is designed to address the underlying molecular cause of FA.

Key Points: 
  • The event will showcase Exicures neuroscience pipeline, including its lead program for Friedreichs Ataxia (FA), XCUR-FXN, which is designed to address the underlying molecular cause of FA.
  • Exicure will present new and previously unreleased preclinical data and discuss progress with XCUR-FXN, which is on track for IND filing in late Q4 2021.
  • Additionally, Exicure will discuss its perspective on XCUR-FXNs competitive differentiation in FA and the programs path to clinical validation.
  • Also, Exicure will provide an update on its expanding pipeline across several rare neurodegenerative diseases of high unmet need and highlight progress with two preclinical programs targeting SCN9A (Nav1.7) for neuropathic pain and CLN3 for Batten Disease.

Reata Announces that The FDA Has Asked The Company to Request a Pre-NDA Meeting for Omaveloxolone for the Treatment of Friedreich’s Ataxia

Retrieved on: 
Wednesday, May 19, 2021
Branches of biology, Frataxin, Proteins, Trinucleotide repeat expansion, Ataxin, Omaveloxolone, Trinucleotide repeat disorder, NFE2L2, Friedreich's ataxia

The Division suggested that the Company withdraw the current meeting request for a Type C meeting and instead request a pre-NDA meeting, which the Division will grant upon receipt.

Key Points: 
  • The Division suggested that the Company withdraw the current meeting request for a Type C meeting and instead request a pre-NDA meeting, which the Division will grant upon receipt.
  • FA is typically caused by a trinucleotide repeat expansion in the first intron of the frataxin gene, which encodes the mitochondrial protein frataxin.
  • The FDA has granted Orphan Drug designation to omaveloxolone for the treatment of Friedreich\xe2\x80\x99s ataxia.
  • Reata possesses exclusive, worldwide rights to develop, manufacture, and commercialize omaveloxolone, and our other next-generation Nrf2 activators.