FXN

Muscular Dystrophy Association and Friedreich’s Ataxia Research Alliance Announce Collaborative Research Grant Using Novel Gene Editing Technology to Address Root Cause of Friedreich’s Ataxia Disease

Retrieved on: 
Wednesday, April 17, 2024
Pillar, Enzyme, Friedreich's ataxia, Gene editing, CRISPR, GAA, Hope, Muscular dystrophy, Ataxia, FXN, MDA, Diabetes, FARA, Muscular Dystrophy Association, Column, RNA, Doctor of Philosophy, Research, Scoliosis, Parent, Schnucks, University of Massachusetts, Disease, Therapy, Biomedical Research, Nikolaus Friedreich, Fatigue, Movement, FA, Art, Genome, Organization, Pharmaceutical industry

This funding will further research into using novel genetic technologies to treat Friedreich’s ataxia (FA).

Key Points: 
  • This funding will further research into using novel genetic technologies to treat Friedreich’s ataxia (FA).
  • The grant, Paired Prime Editors to treat Friedreich’s Ataxia, involves prime editing (PE), a next-generation CRISPR gene editing tool that can precisely target the removal of the GAA expansions in the frataxin (FXN) gene.
  • “Our team of investigators is excited to bring multi-disciplinary expertise to the unique challenges of developing PE for FA.
  • This treatment method being targeted – prime editing – aims to directly address the cause of the disease, which is the GAA expansion in the FXN gene.

Design Therapeutics Outlines Progress Across GeneTAC™ Platform and Announces Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Tuesday, March 19, 2024
Research, Observational study, Cerebellar ataxia, Biotechnology, Gene expression, FXN, Disease, Webcast, Patient, G&A, DM1, Design, IND, Treatment, Clinical trial, Investigational New Drug, Therapy, Completeness, Evaluation, DMPK, FA, HD, Food, FDA, GLP, HTT, Pharmaceutical industry

R&D Expenses: Research and development (R&D) expenses were $11.0 million for the quarter ended December 31, 2023, and $57.1 million for the year ended December 31, 2023.

Key Points: 
  • R&D Expenses: Research and development (R&D) expenses were $11.0 million for the quarter ended December 31, 2023, and $57.1 million for the year ended December 31, 2023.
  • G&A Expenses: General and administrative (G&A) expenses were $4.1 million for the quarter ended December 31, 2023, and $21.1 million for the year ended December 31, 2023.
  • Net Loss: Net loss was $11.8 million for the quarter ended December 31, 2023, and $66.9 million for the year ended December 31, 2023.
  • Cash Position and Operating Runway: Cash, cash equivalents and marketable securities were $281.8 million as of December 31, 2023.

Lexeo Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Operational Highlights

Retrieved on: 
Monday, March 11, 2024
Fast Track, Cohort, Research, Orphan drug, FDA, Cardiovascular disease, Treatment, Clinical trial, Exercise, FTD, ODD, Phenotype, Patient, Public expenditure, Hypertrophy, FXN, Heart, Cadaver, G&A, Development, FA, Pharmaceutical industry

NEW YORK, March 11, 2024 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc. (Nasdaq: LXEO), a clinical stage genetic medicine company dedicated to pioneering treatments for genetically defined cardiovascular diseases and APOE4-associated Alzheimer’s disease, today reported fourth quarter and full year 2023 financial results and provided operational highlights.

Key Points: 
  • Data to date suggest that FA patients may have lower FXN levels in the heart versus peripheral tissues.
  • Across all three cardiac biopsies, we observed an increase in FXN levels as measured by liquid chromatography mass spectrometry relative to pre-treatment baseline levels.
  • Lexeo anticipates the gross proceeds from the private placement to be approximately $95.0 million, before deducting any offering related expenses.
  • G&A expenses were $15.4 million for the year ended December 31, 2023, compared to $12.0 million for the same period in 2022.

Voyager Therapeutics Reports Fourth Quarter and Full Year 2023 Financial and Operating Results

Retrieved on: 
Wednesday, February 28, 2024
Conference call, ALS, Research, Messenger, Telephone, HD, Growth, Exercise, Conference, SMA, SOD1, Alexion, Biomarker, Neurocrine Biosciences, RNA, Therapy, Ataxia, Patient, Neurocrine, FXN, G&A, AAV, DSM-IV codes, PET, Spinal muscular atrophy, Mouse, Brain, Pharmaceutical industry

LEXINGTON, Mass., Feb. 28, 2024 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today reported fourth quarter and full year 2023 financial and operating results.

Key Points: 
  • ET today -
    LEXINGTON, Mass., Feb. 28, 2024 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today reported fourth quarter and full year 2023 financial and operating results.
  • Novartis agreed to pay Voyager $80 million of consideration up front and $20 million for the purchase of newly issued equity in Voyager.
  • Collaboration Revenues: Voyager had collaboration revenue of $90.1 million for the fourth quarter of 2023, compared to $(1.6) million for the same period in 2022.
  • ET to discuss the fourth quarter and full year 2023 financial and operating results.

Voyager Therapeutics Announces Selection of Gene Therapy Development Candidate for Friedreich’s Ataxia in Collaboration with Neurocrine Biosciences, Triggering Milestone Payment

Retrieved on: 
Monday, February 26, 2024
Traffic barrier, ALS, SOD1, Neurocrine Biosciences, Therapy, Neurocrine, FXN, GBA1, AAV, Research, FA, Vaccine

The candidate combines a frataxin (FXN) gene replacement payload with an intravenously administered, blood-brain barrier penetrant, novel capsid derived from Voyager’s TRACER™ capsid discovery platform.

Key Points: 
  • The candidate combines a frataxin (FXN) gene replacement payload with an intravenously administered, blood-brain barrier penetrant, novel capsid derived from Voyager’s TRACER™ capsid discovery platform.
  • Selection of the development candidate triggered a $5 million milestone payment to Voyager, which the Company expects to receive in the first quarter of 2024.
  • Voyager is eligible to receive additional future development and commercialization milestone payments based on the further advancement of this program.
  • We believe our strategy to replace the defective frataxin gene could address the underlying disease etiology of FA.

Nu Holdings Ltd. Reports Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Thursday, February 22, 2024
Professional Services, Payments, Technology, Finance, Fintech, Banking, Analysis, Investment, Holding, ROE, Net income, Yoy, Appendix, Webcast, Acceleration, FX, CSA, Ratio, Result, Security (finance), NU, International Financial Reporting Standards, International, Asset quality, Canadian Aviation Regulations, INSS, Conference, Central Bank of Brazil, IEP, NPL, Insurance, Prospectus, Annual report, Brazilian real, FXN, Bank

Nu Holdings Ltd. (NYSE: NU), (“Nu” or the “Company”), one of the world’s largest digital financial services platforms, released today its Fourth Quarter and Full Year 2023 financial results.

Key Points: 
  • Nu Holdings Ltd. (NYSE: NU), (“Nu” or the “Company”), one of the world’s largest digital financial services platforms, released today its Fourth Quarter and Full Year 2023 financial results.
  • Financial results are expressed in U.S. dollars and are presented in accordance with International Financial Reporting Standards (IFRS).
  • In Brazil, the customer base reached 87.8 million by December 31, 2023, representing 53% of the country’s adult population.
  • Nu is the fourth-largest financial institution by number of customers in the country, according to Brazilian Central Bank data.

Larimar Therapeutics Reports Positive Top-line Data from Phase 2 Dose Exploration Study from 25 mg and 50 mg Cohorts of Nomlabofusp in Patients with Friedreich’s Ataxia

Retrieved on: 
Monday, February 12, 2024
Pharmacology, QOD, FDA, BLA, Skin, Safety, Therapy, Pharmacokinetics, Webcast, Patient, OLE, FXN, Disease, Tissue, FA, Pharmaceutical industry

Participants in the 25 mg (n=13) and 50 mg (n=15) cohorts were randomized 2:1 to receive subcutaneous injections of nomlabofusp or placebo.

Key Points: 
  • Participants in the 25 mg (n=13) and 50 mg (n=15) cohorts were randomized 2:1 to receive subcutaneous injections of nomlabofusp or placebo.
  • Further dose expansion in the OLE will be considered based on safety, pharmacokinetics, and tissue FXN levels from the 25 mg dose of nomlabofusp.
  • All treated patients demonstrated increases in FXN levels in skin cells and the majority of patients also demonstrated increases in FXN levels in buccal cells.
  • Median baseline tissue FXN levels in skin cells were 3.70 pg/µg and 2.12 pg/µg for the 25 mg and 50 mg cohorts, respectively, and in buccal cells were 1.78 pg/µg and 1.61 pg/µg for the 25 mg and 50 mg cohorts, respectively.

Flash News: OKX Wallet Launches f(x) Event Bonus Offering Users who Stake fETH Up to 20% FXN Rewards

Retrieved on: 
Tuesday, January 2, 2024
OKX, Investment, Decentralized finance, FXN, Web3, Cryptocurrency

OKX Wallet is pleased to announce that, until February 26, 2024, users who stake fETH in the f(x) Bonus pool can earn a bonus of up to 20% in FXN rewards.

Key Points: 
  • OKX Wallet is pleased to announce that, until February 26, 2024, users who stake fETH in the f(x) Bonus pool can earn a bonus of up to 20% in FXN rewards.
  • Access the event through the OKX App or the OKX Web3 official website.
  • Only investments made through OKX DeFi following the commencement of the event will be eligible for rewards.
  • OKX DeFi is a powerful DeFi platform that supports earning and staking on about 70 protocols across more than 10 chains.

Nu Holdings Ltd. Reports Third Quarter 2023 Financial Results

Retrieved on: 
Tuesday, November 14, 2023
Professional Services, Payments, Technology, Finance, Fintech, Banking, Social, Investment, Brazilian real, Conference, Analysis, Holding, Average cost method, Webcast, Ratio, International expansion of Netflix, ROE, Security (finance), International, NU, FX, INSS, Financial condition report, Result, Capital adequacy ratio, Acceleration, Insurance, IEP, Prospectus, Central Bank of Brazil, Asset quality, NPL, NYSE, Yoy, Financial result, Pix, Annual report, Phase 10, FXN, International Financial Reporting Standards, Cryptocurrency, Bank, Video game, Audit, Nu, Growth, Nubank, Payroll

Nu Holdings Ltd. (NYSE: NU), (“Nu” or the “Company”), one of the world’s largest digital financial services platforms, released today its Third Quarter 2023 financial results.

Key Points: 
  • Nu Holdings Ltd. (NYSE: NU), (“Nu” or the “Company”), one of the world’s largest digital financial services platforms, released today its Third Quarter 2023 financial results.
  • Financial results are expressed in U.S. dollars and are presented in accordance with International Financial Reporting Standards (IFRS).
  • Below are the Q3’23 performance highlights of Nu Holdings Ltd.:
    Customer growth: Nu added 5.4 million customers in Q3’23 and 18.7 million year-over-year (YoY), reaching a total of 89.1 million customers globally by September 30, 2023.
  • Nu is the fourth-largest financial institution by number of customers in the country, according to Brazilian Central Bank data.

Voyager Therapeutics Reports Third Quarter 2023 Financial and Operating Results

Retrieved on: 
Monday, November 6, 2023
Research, TRACER, Ataxia, IND, SOD1, Alexion, ALS, Toxicology, GBA1, Neurocrine, G&A, Telephone, FXN, Conference, Neurocrine Biosciences, Capsid, Pfizer, Growth, Novartis, DSM-IV codes, GLP, Neurology, IND Culver Line, Conference call, Therapy, D, Good laboratory practice, Pharmaceutical industry, Natural gas, Voyager

LEXINGTON, Mass., Nov. 06, 2023 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to breaking through barriers in gene therapy and neurology, today reported third quarter 2023 financial and operating results.

Key Points: 
  • ET today -
    LEXINGTON, Mass., Nov. 06, 2023 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to breaking through barriers in gene therapy and neurology, today reported third quarter 2023 financial and operating results.
  • SOD1 gene therapy program for amyotrophic lateral sclerosis (ALS): Voyager expects to identify a lead development candidate in 2023 to support an IND in mid-2025.
  • Collaboration Revenues: Voyager had collaboration revenue of $4.6 million for the third quarter of 2023, compared to $41.1 million for the same period in 2022.
  • ET to discuss the third quarter 2023 financial and operating results.