C9orf72

Dewpoint Therapeutics Awarded Target ALS Grant for Development of C-mods for ALS

Retrieved on: 
Tuesday, January 16, 2024
Pathology, Patient, Target, Research, ALS, Dew point, ALS Association, C9orf72, Biotechnology, Science, Repeat, Jackson Laboratory, Mutation, Epistasis, Disease, Therapy, Neurodegenerative disease, Pharmaceutical industry

Challenges to develop effective therapeutics for ALS stem, in part, from the diverse, and poorly understood genetic background of the patients.

Key Points: 
  • Challenges to develop effective therapeutics for ALS stem, in part, from the diverse, and poorly understood genetic background of the patients.
  • Dewpoint leverages an aberration that occurs in ~97% of all ALS patients – independent of genetic background – to drive the discovery and development of a new, and potentially more effective class of therapeutics.
  • “Receiving the Target ALS grant is a tremendous honor and a significant validation for Dewpoint’s novel, therapeutic approach to target condensatopathies,” said Ameet Nathwani, CEO of Dewpoint Therapeutics.
  • Dr. Amy Easton, Sr. Director of Scientific Programs at Target ALS said "Target ALS is excited to fund the generation of in vivo target validation data to support development of Dewpoint Therapeutics’ clinical candidate for ALS.

ALS Finding a Cure, The ALS Association Partner to Reduce Time to Initial ALS Diagnosis

Retrieved on: 
Thursday, August 17, 2023
Massachusetts General Hospital, Trinity College, Neuron, Partnership, Drug development, Speech, Electroencephalography, Spinal cord, C9orf72, Quality of life, RNA, Research, Brain, Cell, Biomarker, Cytoplasm, Messenger, ALS Association, Brain cell, Harvard Medical School, Trinity College Dublin, The, Protein, Johns Hopkins University, Patient, Mass, Risk, EEG, Blood, Cancer, Medicine, Neurology, CSF, Diagnosis, Hospital, Early, ALS, Gene, Peptide, Disease, Pharmaceutical industry, Vaccine, Medical imaging, Management

BOSTON and BOYNTON BEACH, Fla., Aug. 17, 2023 /PRNewswire/ -- ALS Finding a Cure® (ALSFAC) and The ALS Association announced today seven new grants worth a total of $2 million to support the development of early diagnostics for ALS.

Key Points: 
  • To receive an ALS diagnosis, patients must first undergo a battery of tests designed to rule out other conditions.
  • This is highly problematic because it can take a year – or sometimes even longer – for a person with ALS to receive the correct diagnosis.
  • To address this challenge, ALSFAC launched the Early ALS Diagnostics Initiative to identify promising molecular, digital, imaging, and electrophysiological diagnostic approaches for ALS.
  • After receiving a Partnership Grant from The ALS Association, ALSFAC was able to increase the number of projects supported by the initiative and expand its reach internationally.

BenevolentAI Progresses BEN-34712 for the Potential Treatment of ALS into IND-Enabling Studies

Retrieved on: 
Monday, June 5, 2023
Research, Technology, Neurology, Clinical Trials, Software, Biotechnology, Pharmaceutical, Health, Science, Artificial Intelligence, Central nervous system, University, Neurodegenerative disease, C9orf72, CNS, BAI, Oxidative stress, Euronext, Senior lecturer, Patient, Translational neuroscience, Sheffield Institute of Arts Gallery, ALS, IND, Pharmaceutical industry

BenevolentAI ​​(Euronext Amsterdam: BAI), a leader in the development of cutting-edge AI that accelerates biopharma discovery, announces the successful delivery of its pre-clinical candidate for the potential treatment of amyotrophic lateral sclerosis (ALS), BEN-34712.

Key Points: 
  • BenevolentAI ​​(Euronext Amsterdam: BAI), a leader in the development of cutting-edge AI that accelerates biopharma discovery, announces the successful delivery of its pre-clinical candidate for the potential treatment of amyotrophic lateral sclerosis (ALS), BEN-34712.
  • BEN-34712 is an oral, potent and selective brain penetrant RARɑβ (retinoic acid receptor alpha beta) biased agonist and will now enter investigational new drug (IND)-enabling studies.
  • Impaired retinoic acid signalling has been shown to result in neuroinflammation, oxidative stress and mitochondrial dysfunction, all hallmarks of ALS.
  • Anne Phelan, Chief Scientific Officer, BenevolentAI, said: “There remains a significant and urgent need for new and alternative therapies for patients with ALS.

Locanabio Presents Preclinical Data from its Vectorized snRNA Exon Skipping Program for DMD and Cas13d Multi-targeting Program for C9orf72 ALS at the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting

Retrieved on: 
Tuesday, May 16, 2023
Drug development, Duchenne muscular dystrophy, Therapy, ADAR, HRE, Short interspersed nuclear element, Spinal cord, C9orf72, Diaphragm, RNA, Toxicity, Brain, AAV, Messenger, Facial muscles, Model, DMD, SnRNA-seq, Patient, SAN, Reading, Frontotemporal dementia, DPR, Poster, Small nuclear RNA, Exon, ALS, Treatment, Disease, Vaccine, Publication

SAN DIEGO, May 16, 2023 (GLOBE NEWSWIRE) -- Locanabio, Inc., a genetic medicines company developing RNA-targeted therapeutics for patients with rare genetic neuromuscular and neurodegenerative diseases, announced the presentation of data from its lead vectorized snRNA exon skipping program, LBIO-115, in development for the treatment of Duchenne muscular dystrophy (DMD). A second presentation highlights data from the company’s Cas13d multi-targeting program for C9orf72-related amyotrophic lateral sclerosis (ALS). The ASGCT 26th Annual Meeting is being held May 16-20, in Los Angeles.

Key Points: 
  • A second presentation highlights data from the company’s Cas13d multi-targeting program for C9orf72-related amyotrophic lateral sclerosis (ALS).
  • The poster describes data that demonstrate high levels (>90%) of exon 51 skipping and dystrophin restoration in human DMD skeletal and cardiac muscle cells after LBIO-115 treatment.
  • In a relevant in vivo model (del52hDMD/mdx), robust levels of exon 51 skipping were observed at four weeks post-intramuscular administration of LBIO-115.
  • Dr. Burns added, “Existing early clinical studies in DMD patients with a duplication in exon 2 provided clinical validation for an snRNA-mediated exon skipping approach.

AFTD and ALS Association Launch Funding Opportunity for Digital Tool Development in ALS and Frontotemporal Dementia

Retrieved on: 
Thursday, May 11, 2023
Partnership, Travel, Biotechnology, C9orf72, Pathology, Research, Multimedia, RNA, Person, Movement, ALS Association, The, Lewy body dementias, Diagnosis, Digital, ALS, FTD, Medical imaging, Vaccine, Pharmaceutical industry

KING OF PRUSSIA, Pa., May 11, 2023 /PRNewswire/ -- The Association for Frontotemporal Degeneration (AFTD) and The ALS Association are launching a funding opportunity to support collaborative development of digital assessment tools across two overlapping neurodegenerative disorders: frontotemporal degeneration (FTD) and amyotrophic lateral sclerosis (ALS).

Key Points: 
  • KING OF PRUSSIA, Pa., May 11, 2023 /PRNewswire/ -- The Association for Frontotemporal Degeneration (AFTD) and The ALS Association are launching a funding opportunity to support collaborative development of digital assessment tools across two overlapping neurodegenerative disorders: frontotemporal degeneration (FTD) and amyotrophic lateral sclerosis (ALS).
  • If correctly designed and applied, digital assessment tools have the potential to expand research into, and strengthen care for, ALS and FTD.
  • Once considered separate neurodegenerative disorders, FTD and ALS are now recognized to have many commonalities at the molecular, cellular, and clinical levels.
  • "AFTD is proud to partner with The ALS Association to foster collaboration across the FTD and ALS research communities," Dr. Dacks added.

Wave Life Sciences Reports Fourth Quarter and Full Year 2022 Financial Results and Provides Business Update

Retrieved on: 
Wednesday, March 22, 2023
PRISM, 50/50 (2011 film), Takeda, C9orf72, MBA, Cerebrospinal fluid, Protein, MD, Observation, RNA interference, DSM-IV codes, Disease, Duchenne muscular dystrophy, Therapy, Genetics, Aimer, Pharmacokinetics, Mutation, Research, Gene expression, Small RNA, AATD, N-Acetylgalactosamine, Episcopal conference, Muscular Dystrophy Association, Sirna Therapeutics, Liver, MDA, IND, Conference, Safety, GSK, SERPINA1, Messenger, Webcast, APP, RNA editing, HD, ICV, Central nervous system, Q&A, Genomics, RNA, CNS, CTAS, DMD, Mouse, Pharmaceutical industry, Broadcasting, Vaccine, Cryptocurrency, Video game, Wave

ET today

Key Points: 
  • ET today
    CAMBRIDGE, Mass., March 22, 2023 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases, today announced financial results for the fourth quarter and full year ended December 31, 2022 and provided a business update.
  • Research and development expenses were $31.1 million in the fourth quarter of 2022 as compared to $25.8 million in the same period in 2021.
  • As of December 31, 2022, Wave had $88.5 million in cash and cash equivalents, as compared to $150.6 million as of December 31, 2021.
  • ET to discuss the fourth quarter and full year 2022 financial results and provide a business update.

Alector Reports Fourth Quarter and Full Year 2022 Financial Results and Provides Corporate Update

Retrieved on: 
Tuesday, February 28, 2023
Cerebrospinal fluid, Knowledge, Safety, Alzheimer's disease, Central nervous system, GSK, Alector, ALEC, Glial fibrillary acidic protein, PD, Information technology, OLE, Disease, NACC, C9orf72, TREM2, CSF, Clinical trial, International Conference on Emerging Infectious Diseases, Investment, Patient, LTE, DSM-IV codes, Astrogliosis, Granulin, GFAP, Patent, Plasma, Total, MS4A, Exercise, Microglia, Conference, AbbVie, FTD, AD, PK, Frontotemporal dementia, Arnon Rosenthal, Pharmaceutical industry, Cryptocurrency, Management, Research

As of December 31, 2022, Alector’s cash, cash equivalents and investments totaled $712.9 million.

Key Points: 
  • As of December 31, 2022, Alector’s cash, cash equivalents and investments totaled $712.9 million.
  • Results demonstrated that AL101 was well tolerated and increased progranulin levels in plasma and CSF in a dose-dependent manner.
  • Alector plans to complete INVOKE-2 trial enrollment in the third quarter of 2023, with top-line data expected by the fourth quarter of 2024.
  • Collaboration revenue for the quarter ended December 31, 2022, was $14.4 million, compared to $14.0 million for the same period in 2021.

Alector Reports Third Quarter 2022 Financial Results and Provides Business Update

Retrieved on: 
Tuesday, November 8, 2022
IND, ALEC, Clinical trial, Safety, Immunotherapy, Survival, Standard of care, Cell, Security (finance), Forward-looking statement, Documentation, TREM2, Annual general meeting, Total, Society for Immunotherapy of Cancer, Global Health Innovative Technology Fund, SEC, Alector, U.S. Securities and Exchange Commission, Pharmacokinetics, Patient, Frontotemporal dementia, Private Securities Litigation Reform Act, Arnon Rosenthal, Neurodegeneration, Nasdaq, C9orf72, Microglia, SIGLEC, MS4A, Myelocyte, AD, Poster, Society, AbbVie, PD, GLOBE, Investment, Genetics, GSK, Biomarker, Disease, Immune system, SITC, Pharmaceutical industry, Management, Vaccine, Research

SOUTH SAN FRANCISCO, Calif., Nov. 08, 2022 (GLOBE NEWSWIRE) -- Alector, Inc. (Nasdaq: ALEC), a clinical-stage biotechnology company pioneering immuno-neurology and innate immuno-oncology, today reported third quarter 2022 financial results and recent portfolio and business updates. As of September 30, 2022, Alector’s cash, cash equivalents and investments totaled $758.3 million.

Key Points: 
  • Sara Kenkare-Mitra, Ph.D., President and Head of Research and Development at Alector, added, Our immuno-oncology pipeline continues to advance as well.
  • Peters expertise in the human genetics of neurodegenerative disease is important to our efforts at Alector.
  • Total research and development expenses for the quarter ended September 30, 2022, were $48.3 million, compared to $43.1 million for the quarter ended September 30, 2021.
  • These documents contain and identify important factors that could cause the actual results for Alector to differ materially from those contained in Alectors forward-looking statements.

Research Provides New Insight into ALS

Retrieved on: 
Wednesday, August 24, 2022
Science, Neurology, Biotechnology, Research, Pharmaceutical, Health, Clinical Trials, Other Health, Massachusetts General Hospital, SOD1, Harvard Medical School, Research, Neurotoxicity, Filtration, Cerebrospinal fluid, TARDBP, CSF, Mouse, Apolipoprotein B, C9orf72, Pathology, Hospital, Center, Neurology, International, Tisch, Neurodegeneration, MS, Fals, Spinal cord, Bacillus, Physical, ALS, Patient, Dentistry

Using an innovative experimental approach to deliver cerebrospinal fluid (CSF) from ALS patients, researchers developed a novel CSF-mediated mouse model to investigate pathophysiological mechanisms in different ALS subtypes.

Key Points: 
  • Using an innovative experimental approach to deliver cerebrospinal fluid (CSF) from ALS patients, researchers developed a novel CSF-mediated mouse model to investigate pathophysiological mechanisms in different ALS subtypes.
  • The impact of this research is potentially groundbreaking for patients with sporadic ALS, the predominant form of this devastating disease, said Dr. Saud A. Sadiq, Director and Chief Research Scientist at the Tisch MS Research Center of New York.
  • The patients were seen at the Sean M. Healey and AMG Center for ALS at Massachusetts General Hospital, as well as the Tisch MS Research Center of New Yorks Larry G. Gluck Division of ALS Research.
  • The mission of the Tisch Multiple Sclerosis Research Center of New York is to conduct groundbreaking medical research to ensure unparalleled care and positive outcomes for MS patients.

Intravacc and DZNE awarded EU funding to develop vaccine against genetic ALS variant

Retrieved on: 
Wednesday, July 6, 2022
Diagnosis, C9orf72, Death, German Center for Neurodegenerative Diseases, Patient, Dementia, DPT, Influenza, Research, University, Brain, Bill & Melinda Gates Foundation, Man, Spinal cord, Paralysis, Union, Disease, ALS, Association, DZNE, Parkinson's disease, CDMO, Immune system, Infection, Motor neuron, Woman, Risk, Helmholtz Association, Neurodegeneration, Technology, Antibody, Nervous system, Neuron, Motor neuron disease, Human, Frontotemporal dementia, Pathology, Vaccine, Alzheimer's disease, Vaccination, Movement disorders, Mutation, WHO, Pharmaceutical industry, EU, Haemophilus influenzae

The project aims to develop the vaccine candidate identified at DZNE to the point where it can be clinically tested in humans.

Key Points: 
  • The project aims to develop the vaccine candidate identified at DZNE to the point where it can be clinically tested in humans.
  • About 5-10% of all ALS cases are caused by a mutation in the C9orf72 gene, making it the most common genetic ALS variant.
  • DZNE developed an experimental vaccine that instructs the immune system to produce antibodies against these harmful poly-GA molecules.
  • Clinical trials for C9orf72 ALS, which is the most common genetic variant of ALS, are expected to commence in 2025.