Ataxia

Larimar Therapeutics Reports First Quarter 2024 Operating and Financial Results

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목요일, 5월 9, 2024
FDA, Food and Drug Administration, Safety, Skin, Pharmacokinetics, Food, Commercial, Ataxia, FXN, Research, BioCryst Pharmaceuticals, Spark Therapeutics, ViroPharma, Pharmacist, Hereditary angioedema, OLE, Spark, Senior, Patient, FA, Gene expression, Database, BLA, Interim, Clinical trial, Therapy, Pharmacology, Pharmaceutical industry

In February 2024, Larimar raised net proceeds of approximately $161.8 million through a public offering of common stock.

Key Points: 
  • In February 2024, Larimar raised net proceeds of approximately $161.8 million through a public offering of common stock.
  • In March 2024, Larimar began to build its commercial team with the appointment of Frank Nazzario, RPh, as Vice President of Commercial.
  • Research and development expenses for the first quarter of 2024 were $12.9 million, compared to $4.6 million for the first quarter of 2023.
  • General and administrative expenses were $3.8 million in the first quarter of 2024, compared to $3.1 million in the first quarter of 2023.

Encoded Therapeutics Presents Preclinical Data Across its Gene Therapy Portfolio at the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)

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화요일, 5월 7, 2024
Biotechnology, Neurology, Health, Genetics, Pharmaceutical, DRG, SCN9A, Seizure, Risk, Angelman syndrome, Locomotion, UBE3A, Human, Neurodevelopmental disorder, Abstract, Code, AAV, Epilepsy, Ataxia, CNS, GABAergic, Disease, Incidence, IPSC, Spinal cord, MAPT, Intellectual disability, Central nervous system, DSM-IV codes, Neuron, NHP, Therapy, Gene, MicroRNA, Vaccine

“We are thrilled to share multiple advancements across our portfolio of precision genetic medicines, showcasing substantial progress across various fronts.

Key Points: 
  • “We are thrilled to share multiple advancements across our portfolio of precision genetic medicines, showcasing substantial progress across various fronts.
  • These data underscore our platform’s potential to deliver precision therapies for a range of CNS diseases,” said Stephanie Tagliatela, Chief Scientific Officer at Encoded.
  • Encoded’s vector engineering platform has enabled the development of GABA-selective AAV-mediated gene therapies designed to potentiate GABAergic neurotransmission and modulate the circuit dysfunction underlying refractory epilepsies.
  • These data support further development of our candidate gene therapy for the treatment for refractory epilepsies.

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

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수요일, 4월 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.

Larimar Therapeutics Reports Fourth Quarter and Full Year 2023 Operating and Financial Results and Provides Update on Nomlabofusp Development

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목요일, 3월 14, 2024
Research, Database, Gene expression, Disease, Patient, Accounting, OLE, Insurance, Ataxia, Clinical trial, Skin, Therapy, Safety, Pharmacokinetics, Interim, FA, FDA, BLA, Pharmaceutical industry

BALA CYNWYD, Pa., March 14, 2024 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (“Larimar”) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today reported its fourth quarter and full year 2023 operating and financial results.

Key Points: 
  • BALA CYNWYD, Pa., March 14, 2024 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (“Larimar”) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today reported its fourth quarter and full year 2023 operating and financial results.
  • Nomlabofusp was generally well-tolerated and demonstrated dose-dependent increases in skin and buccal cell frataxin levels.
  • Research and development expenses for the fourth quarter of 2023 were $10.6 million compared to $7.2 million for the fourth quarter of 2022.
  • General and administrative expenses for the fourth quarter of 2023 were $3.5 million compared to $3.2 million for the fourth quarter of 2022.

PacBio Announces PureTarget™ Repeat Expansion Panel, Expanding its Portfolio of End-to-End Clinical Research Solutions

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화요일, 3월 12, 2024
Gene, Methylation, FTD, FMR1, ALS, Associate, Myotonic dystrophy, Abraxis BioScience, Huntington's disease, DNA, Apple IIe, Ataxia, Laboratory, University College London, Frontotemporal dementia, DSM-IV codes, Mosaic, High fidelity, Instability

MENLO PARK, Calif., March 12, 2024 /PRNewswire/ -- PacBio (NASDAQ: PACB), a leading developer of high-quality, highly accurate sequencing solutions, today announced the PureTarget repeat expansion panel, a new solution designed to enable the comprehensive analysis of 20 genes associated with serious neurological disorders, including challenging-to-sequence genes with tandem repeat expansions. The new long-read workflow can minimize iterative analysis using legacy technology, and reduce the time needed to identify disease-causing variants and associated methylation signatures.

Key Points: 
  • "This product uses a differentiated method to do targeted long-read sequencing of DNA in its pure state.
  • Continuing the commitment to deliver comprehensive solutions, PacBio has prioritized delivering end-to-end workflows with their latest products.
  • The PureTarget Repeat Expansion Panel workflow utilizes the recently announced Nanobind PanDNA kit , ensuring robust results optimized for Revio and Sequel IIe users.
  • Customers can multiplex up to 48 samples on the PacBio Revio sequencing system and 24 samples on the PacBio Sequel IIe sequencing system.

Larimar Therapeutics Announces the Dosing of the First Patient in Long-term Open Label Extension Study for Nomlabofusp in Patients with Friedreich’s Ataxia

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월요일, 3월 11, 2024
Pharmacology, Friedreich's ataxia, FDA, BLA, Ataxia, Mitochondrion, Safety, Therapy, Pharmacokinetics, Patient, OLE, FA, Gene expression, Pharmaceutical industry

Nomlabofusp (CTI-1601) is a novel protein replacement therapy designed to address the root cause of Friedreich's ataxia (FA) by delivering frataxin to mitochondria.

Key Points: 
  • Nomlabofusp (CTI-1601) is a novel protein replacement therapy designed to address the root cause of Friedreich's ataxia (FA) by delivering frataxin to mitochondria.
  • “We are pleased to dose the first patient in our OLE study, further advancing the nomlabofusp clinical program and building on the successful completion of our Phase 2 dose escalation study.
  • Based on our Phase 1 and Phase 2 findings, we expect to continue daily dosing throughout the study,” said Carole Ben-Maimon, MD, President, and Chief Executive Officer of Larimar.
  • Initial data from the OLE study is expected in Q4 2024.

Prime Medicine Reports Full Year 2023 Financial Results and Provides Business Updates

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금요일, 3월 1, 2024
Sequence, Composition, Liver, RHO, Immunotherapy, Investment, FDA, Research and development, HSC, Food, CGD, Patent, SCIP, Lung, Ataxia, Cystic Fibrosis Foundation, Hematology, Administration, Patient, Target, IND, AAV, CTA, Pharmaceutical industry

CAMBRIDGE, Mass., March 01, 2024 (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 for the full year ended December 31, 2023 and provided a business update.

Key Points: 
  • “In 2024, we anticipate undergoing a significant transformation, maturing into a clinical-stage company and bringing the first-ever Prime Editing-based therapeutic candidate to patients.
  • This funding will allow Prime Medicine to progress two distinct strategies for applying Prime Editing to treat CF: hotspot editing and PASSIGE™ (Prime Assisted Site Specific Integrase Gene Editing).
  • Gross proceeds to Prime Medicine from the offering, before deducting underwriting discounts and commissions and offering expenses, were approximately $161.0 million.
  • In January 2024, Prime Medicine announced the appointment of Allan Reine, M.D., as the Company’s Chief Financial Officer.

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

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수요일, 2월 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.

Avenue Therapeutics Announces Publication in Drug Development Research Highlighting First-In-Class Preclinical Data of BAER-101 in a Translational Model of Absence Epilepsy

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목요일, 2월 22, 2024
Epilepsy, Pharmacology, DSM-IV codes, Research, GABAA, Drug resistance, 300X, Dizziness, Drug development, PAM, Volunteering, GAERS, Risk, Generalized tonic–clonic seizure, Deficit spending, Rat, Therapy, Ataxia, Memory, MED, Human, Cognition, Patient, Somnolence, Addiction, Partnership, Pharmaceutical industry

MIAMI, Feb. 22, 2024 (GLOBE NEWSWIRE) -- Avenue Therapeutics, Inc. (Nasdaq: ATXI) (“Avenue” or the “Company”), a specialty pharmaceutical company focused on the development and commercialization of therapies for the treatment of neurologic diseases, today announced the publication of preclinical in vivo data in Drug Development Research highlighting BAER-101’s full suppression of seizure activity using the Genetic Absence Epilepsy Rats from Strasbourg (“GAERS”) model of absence epilepsy.

Key Points: 
  • MIAMI, Feb. 22, 2024 (GLOBE NEWSWIRE) -- Avenue Therapeutics, Inc. (Nasdaq: ATXI) (“Avenue” or the “Company”), a specialty pharmaceutical company focused on the development and commercialization of therapies for the treatment of neurologic diseases, today announced the publication of preclinical in vivo data in Drug Development Research highlighting BAER-101’s full suppression of seizure activity using the Genetic Absence Epilepsy Rats from Strasbourg (“GAERS”) model of absence epilepsy.
  • The publication describes the extent of anti-seizure activity of BAER-101 in the GAERS model, a widely used and translationally relevant animal model.
  • The study demonstrated full suppression of seizure activity with a minimal effective dose (MED) of 0.3 mg/kg.
  • “The preclinical data published in Drug Development Research demonstrate BAER-101’s ability to fully suppress seizures in the GAERS model, a translational animal model for anti-seizure drug development with a documented high predictability of response in humans.

Glycomine Announces Encouraging Efficacy Data from Ongoing Phase 2 Clinical Study in PMM2-CDG

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월요일, 3월 4, 2024
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, Rare disease, International, Caregiver, Adolescence, CMO, Acetazolamide, Quality of life, Conference, Ataxia, Patient, PMM2-CDG, Trial of the century

This compares favorably to prior results reported with acetazolamide which demonstrated an average improvement of 6 points after 25 weeks in adult and pediatric patients (n=25).

Key Points: 
  • This compares favorably to prior results reported with acetazolamide which demonstrated an average improvement of 6 points after 25 weeks in adult and pediatric patients (n=25).
  • “We are very encouraged by the initial results we have seen and delighted to be part of this important study.”
    The GLM101-002 Phase 2 clinical study has enrolled 10 adult patients in the U.S. and Spain (ClinicalTrials.gov Identifier: NCT05549219).
  • Study participants have received GLM101 at either 10 mg/kg (n=3), 20 mg/kg (n=3), or 30 mg/kg (n=4) for up to 24 weeks.
  • Four adolescent patients have initiated treatment, and the study is planned to be extended into younger PMM2-CDG patients in the coming months.