Amyotrophic lateral sclerosis

AC Immune Awarded New Grants from MJFF and Target ALS Supporting Programs Targeting TDP-43

Retrieved on: 
Tuesday, February 7, 2023
Amyotrophic lateral sclerosis, Barrow Neurological Institute, Target, Alpha-synuclein, Disease, Research, Diagnosis, Neurodegeneration, Pathology, The Michael J. Fox Foundation, NDD, MJFF, Frontotemporal lobar degeneration, Precision medicine, International Center, PET, FTLD, Patient, Drug discovery, ALS, Kansas City University, National Center for Research Resources, Senior, AC, TAR, Engineering, Pharmaceutical industry, Medical imaging, Biotechnology

Fox Foundation for Parkinson’s Research (MJFF) and Target ALS Foundation (Target ALS) supporting research programs to enable diagnosis of TDP-43 (TAR DNA-binding protein 43), recognized as an important target in multiple neurodegenerative diseases (NDDs) such as amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD) and as a prominent co-pathology in Alzheimer’s and Parkinson’s diseases.

Key Points: 
  • Fox Foundation for Parkinson’s Research (MJFF) and Target ALS Foundation (Target ALS) supporting research programs to enable diagnosis of TDP-43 (TAR DNA-binding protein 43), recognized as an important target in multiple neurodegenerative diseases (NDDs) such as amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD) and as a prominent co-pathology in Alzheimer’s and Parkinson’s diseases.
  • Dr. Andrea Pfeifer, CEO of AC Immune SA, commented: “It is an honor to have the support of MJFF and Target ALS, two leading international organizations that recognize the pressing need for diagnostics to detect pathological TDP-43.
  • AC Immune and its collaborators recently demonstrated their expertise in developing cutting-edge PET imaging agents by providing the first images of alpha-synuclein.
  • The development of a TDP-43 specific biofluid-based diagnostic test has the potential to more rapidly enable confirmed early diagnosis.”
    The MJFF and Target ALS grants collectively provide more than USD 500,000 in additional non-dilutive capital to support the advancement of diagnostic programs targeting TDP-43.

Ashvattha Therapeutics Announces First Patient Enrolled in a Phase 1 Study of [18F]OP-801 for Use as Imaging Agent

Retrieved on: 
Monday, February 6, 2023
Amyotrophic lateral sclerosis, Disease, Stanford University School of Medicine, Safety, HD, Trial of the century, Neuroinflammation, Pharmacokinetics, Microglia, CT, Neurodegeneration, Patient, HDT, Clinical trial, ALS, Precision medicine, Doctor of Philosophy, Brain, Therapy, Inflammation, Medical imaging

REDWOOD CITY, Calif., Feb. 06, 2023 (GLOBE NEWSWIRE) -- Ashvattha Therapeutics (“Ashvattha”), a clinical-stage company developing novel nanomedicines called hydroxyl dendrimer therapeutics (HDT), today announced that the first patient has been enrolled in a Phase 1/2 clinical study to evaluate the safety, pharmacokinetics and biodistribution of an intravenously administered dose of [18F]OP-801 in healthy volunteers and in patients with amyotrophic lateral sclerosis (ALS) (NCT05395624). [18F]OP-801 is a hydroxyl dendrimer (HD) imaging agent that selectively targets reactive macrophages and microglia – key markers of neuroinflammation.

Key Points: 
  • [18F]OP-801 is a hydroxyl dendrimer (HD) imaging agent that selectively targets reactive macrophages and microglia – key markers of neuroinflammation.
  • “The initiation of the clinical trial evaluating [18F]OP-801 as an imaging agent is a significant step to unlocking the potential of our proprietary HD technology for treatment of neurological diseases,” said Jeffrey Cleland, Ph.D., chairman, CEO and president of Ashvattha Therapeutics.
  • “With [18F]OP-801, we will be able to estimate HDT uptake in the diseased part of the brain prior to treating patients with the HDT.
  • The study will also measure the biodistribution and clearance of the HD imaging agent in both populations and evaluate the uptake of [18F]OP-801 in regions of neuroinflammation in ALS patients using PET/computed tomography (CT) scans.

uniQure and Apic Bio enter into global licensing agreement for APB-102, a clinical stage gene therapy for patients with ALS caused by mutations in SOD1

Retrieved on: 
Tuesday, January 31, 2023
Amyotrophic lateral sclerosis, Mouse, Food, IND, Neuron, Motor neuron disease, Survival, Mirna, Patient, ALS, Doctor of Philosophy, Research, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Pharmaceutical industry, Vaccine, Financial services, SOD1

LEXINGTON, Mass. and AMSTERDAM and CAMBRIDGE, Mass., Jan. 31, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, and Apic Bio, an innovative gene therapy company developing novel treatment options for patients with rare genetic diseases, today announced that they have entered into a global licensing agreement for APB-102 to treat superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS), a rare, genetic form of ALS. Under the agreement, uniQure acquires global rights for the development and commercialization of APB-102, adding to its pipeline of gene therapies to treat neurological disorders. The U.S. Food and Drug Administration has cleared the investigational new drug (IND) application for APB-102 and has granted Orphan Drug and Fast Track designations.

Key Points: 
  • Under the agreement, uniQure acquires global rights for the development and commercialization of APB-102, adding to its pipeline of gene therapies to treat neurological disorders.
  • The U.S. Food and Drug Administration has cleared the investigational new drug (IND) application for APB-102 and has granted Orphan Drug and Fast Track designations.
  • Mutations in the SOD1 gene of ALS account for approximately one-fifth of all inherited forms of this fatal disease1.
  • Relevant SOD1 reduction in spinal cord motor neurons also was demonstrated in rodents, as well as in non-human primates at proposed clinical doses.

Cytokinetics Announces Recipients of Its Fifth Annual Communications Grant Program

Retrieved on: 
Monday, January 30, 2023
Amyotrophic lateral sclerosis, HCM, Engagement, Disease, Education, Diagnosis, Heart transplantation, Heart, National Coalition for Syrian Revolutionary and Opposition Forces, Cardiomyopathy, Chapter, Quality of life, Hypertrophic cardiomyopathy, Mended Hearts, Cytokinetics, Patient, SAN, Health equity, Cardiovascular disease, Patient advocacy, Woman, MHI, ALS, Organization, Risk, Heart failure, ARC, Pharmaceutical industry, Medical device

SOUTH SAN FRANCISCO, Calif., Jan. 30, 2023 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) today announced the recipients of its fifth annual Communications Grant Program, intended to support communications, awareness building and community engagement for nonprofit organizations serving the patient community.

Key Points: 
  • SOUTH SAN FRANCISCO, Calif., Jan. 30, 2023 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) today announced the recipients of its fifth annual Communications Grant Program, intended to support communications, awareness building and community engagement for nonprofit organizations serving the patient community.
  • The grants will support projects to increase organizational reach, launch educational programs, grant patients access to conferences and reach underserved populations.
  • “We are continuously impressed by the quality of proposals we receive, and this year’s recipients were particularly impactful, reflecting the unique needs of the patient communities they serve,” said Mary Pomerantz, Cytokinetics’ Senior Director of Patient Advocacy and Engagement.
  • HeartBrothers will use this grant to expand outreach efforts to grow digital and in-person partnerships with heart failure hospitals nationwide and increase awareness and access to its resources for patients.

New Approaches to Treating Infections in Cystic Fibrosis Patients - AR 501 Phase 2 Trial Update

Retrieved on: 
Tuesday, January 17, 2023
FCCP, Tripler Army Medical Center, EST, Cystic fibrosis, MHS, Public health, Amyotrophic lateral sclerosis, Madigan Army Medical Center, Epidemiology, Johns Hopkins Bloomberg School of Public Health, Degenerative disease, Pediatrics, Patient, KOL, CF, Johns Hopkins School of Medicine, Medicine, Doctor of Philosophy, ALS, National Jewish Health, Pharmaceutical industry, MD, Mycobacterium

Los Gatos, Calif., Jan. 17, 2023 (GLOBE NEWSWIRE) -- Aridis Pharmaceuticals, Inc. (NASDAQ: ARDS), a biopharmaceutical company focused on the discovery and development of novel anti-infective therapies for treating life-threatening infections, today announced its participation in Key Opinion Leader (KOL) event focusing on the anti-infective treatment landscape in cystic fibrosis (CF) and the Company’s Phase 2a trial of AR-501 in CF patients. AR-501 is an inhaled formulation of gallium citrate with broad-spectrum anti-infective activity being developed to treat chronic lung infections in CF patients. The KOL event will take place on January 24 at 11 a.m. EST.

Key Points: 
  • AR-501 is an inhaled formulation of gallium citrate with broad-spectrum anti-infective activity being developed to treat chronic lung infections in CF patients.
  • The KOL event will take place on January 24 at 11 a.m. EST.
  • The virtual event will feature key opinion leaders Jane E. Gross, MD, PhD, and Noah Lechtzin, MD, MHS, FCCP.
  • Carl Byrnes, Managing Director and Senior Research Analyst at Northland Capital Markets will host and moderate the virtual KOL.

WILLOW ANNOUNCES FOLLOW-ON ENGAGEMENT FOR NEXT PHASE OF PARTNERED PHARMACEUTICAL PROJECT

Retrieved on: 
Tuesday, January 17, 2023
API, WLLW, Sandhill, TUDCA, Active ingredient, Amyotrophic lateral sclerosis, Cannabinoid, TSX, Tauroursodeoxycholic acid, Organic acid, UDCA, Primary sclerosing cholangitis, Willow, Growth, Research, Ursodeoxycholic acid, ALS, Fine chemical

This represents the continuation of the project, the first phase of which the Company announced May 31, 2022.

Key Points: 
  • This represents the continuation of the project, the first phase of which the Company announced May 31, 2022.
  • Sandhill is partnered with a large-cap, multi-national healthcare company and the targeted product is manufacture of the large volume API, ursodeoxycholic acid (UDCA).
  • In this next phase of development, the Company anticipates several hundred thousand U.S. dollars in research fees, along with a milestone payment later this year for initial production batches.
  • Upon successful completion, it is expected that Willow's proprietary, optimized enzyme will be taken toward commercialization for the end market active pharmaceutical ingredients ("APIs"), where Willow will receive additional milestone and production-based royalty payments.

Eclipsebio Announces Publication of New Method to Optimize and Scale the Detection of RNA-Binding Protein Sites to Accelerate the Understanding of RNA Regulation

Retrieved on: 
Tuesday, January 17, 2023
Oncology, Health, Hospitals, Genetics, General Health, Pharmaceutical, Biotechnology, Eclipse, RBP, Immunoprecipitation, Disease, ABC, RT, RNA-binding protein, Amyotrophic lateral sclerosis, DNA, NASA eClips, University of California, San Diego, Protein, Therapy, Genomics, RNA, Research, Cancer, IP, UC, Vaccine, Television, Nature Methods

The study, published in Nature Methods , was led by Eclipsebio’s Associate Director of Research Daniel Lorenz, Ph.D., and co-founder Gene Yeo, Ph.D.

Key Points: 
  • The study, published in Nature Methods , was led by Eclipsebio’s Associate Director of Research Daniel Lorenz, Ph.D., and co-founder Gene Yeo, Ph.D.
  • Enhanced cross-linking immunoprecipitation followed by high-throughput sequencing, or eCLIP , is a powerful technique to uncover RNA sequences where RNA-binding proteins (RBPs) bind, known as RBP interactomes.
  • RBPs regulate RNA molecules, controlling the function and quantity of proteins expressed in a cell.
  • We look forward to supporting important RBP research with our current and future eCLIP products that will include the ABC technology.”

Amylyx Pharmaceuticals Announces Exclusive AMX0035 Distribution Agreement with Israel-Based Neopharm

Retrieved on: 
Tuesday, January 17, 2023
Biotechnology, General Health, Neurology, Health, Pharmaceutical, Spinal cord, Brain, Amyotrophic lateral sclerosis, Medication, Failure rate, ALS, National Defense Authorization Act, Pharmaceutical industry

Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”) today announced it has entered into an exclusive license and distribution agreement with Neopharm in which Neopharm will commercialize, subject to regulatory review and approval, AMX0035 (sodium phenylbutyrate and ursodoxicoltaurine) for the treatment of amyotrophic lateral sclerosis (ALS) in Israel, Gaza, West Bank, and the Palestinian Authority.

Key Points: 
  • Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”) today announced it has entered into an exclusive license and distribution agreement with Neopharm in which Neopharm will commercialize, subject to regulatory review and approval, AMX0035 (sodium phenylbutyrate and ursodoxicoltaurine) for the treatment of amyotrophic lateral sclerosis (ALS) in Israel, Gaza, West Bank, and the Palestinian Authority.
  • “This agreement with Neopharm is an important step towards expanding AMX0035’s availability in the EMEA (Europe, Middle East, and Africa) region, if approved,” said Stéphanie Hoffmann-Gendebien, General Manager and Head of EMEA at Amylyx.
  • “The addition of AMX0035 to our portfolio and the opportunity to serve the ALS community in Israel and beyond is an honor,” said Efi Shnaidman, General Manager of Neopharm Israel.
  • “We look forward to working with Amylyx to serve people living with ALS who may benefit from this important new treatment option.”
    Under the terms of the agreement, Neopharm will receive exclusive rights to commercialize AMX0035 in the covered territory and will be responsible for all regulatory filings and obligations required for the registration and reimbursement of AMX0035.

LinusBio Raises $16 Million to Scale Growth and Deliver Tangible Outcomes

Retrieved on: 
Thursday, January 12, 2023
Mental Health, Managed Care, Medical Devices, General Health, Hospitals, Neurology, Science, Genetics, Clinical Trials, FDA, Biotechnology, Health, Health Technology, Other Science, Research, Biometrics, Oncology, Partner (business rank), Psychosis, Farmer, Union, Entrepreneurship, Francisco Partners, Cancer, Physiology, Factor X, Autism, White House, Degenerative disease, ALS, General partnership, CE marking, Amyotrophic lateral sclerosis, Hair, PECASE, Environment, Biomarker, FDA, Organization, Diagnosis, Inflammatory bowel disease, Genomics, Investment, DRS, Science, White, Gastroenterology, Kidney, Farmers Business Network, Schizophrenia, Medical imaging, Medicine, Salesforce

It is the first technology platform that maps the molecular dynamics of human physiology in a time-dependent manner.

Key Points: 
  • It is the first technology platform that maps the molecular dynamics of human physiology in a time-dependent manner.
  • “Our mission is simple: to deliver better outcomes for people and families affected by complex health conditions,” said Manish Arora, PhD., Co-Founder and CEO of LinusBio.
  • “LinusBio has the opportunity to fundamentally transform the healthcare industry.”
    Arora, the CEO of LinusBio, is an environmental epidemiologist and exposure biologist.
  • The funding will be used to grow the LinusBio team, deliver more positive outcomes across more health conditions, and develop a world-class platform that will serve as a central hub for diagnostics and treatment.

Xlife Sciences portfolio company launches project to research a potential therapy for amyotrophic lateral sclerosis (ALS)

Retrieved on: 
Thursday, February 2, 2023
Disease, Muscle atrophy, Degenerative disease, Speech, Science, Motor neuron, Amyotrophic lateral sclerosis, Procaine, PD, Pharmacotherapy, Woman, Nervous system, University Medical Center, Annual report, Patient, Neurology, Research, Department of Neurology, Xuanwu hospital, Georgetown University Medical Center, Liver, ALS, Pharmaceutical industry, Dentistry, Clothing, Riluzole

Xlife Sciences' (SIX: XLS) portfolio company inflamed pharma has launched a research project to develop a potential therapy for amyotrophic lateral sclerosis (ALS).

Key Points: 
  • Xlife Sciences' (SIX: XLS) portfolio company inflamed pharma has launched a research project to develop a potential therapy for amyotrophic lateral sclerosis (ALS).
  • ALS is a chronically progressive, degenerative disease of the nervous system for which there are only symptomatic treatment options so far.
  • [2]
    Frank Plöger, Chief Scientific Officer of Xlife Sciences, comments: “We are looking forward to working with Jan Koch and his team.
  • Together, we hope to lay the scientifically sound, preclinical foundation for a potential drug therapy for the treatment of ALS”.