Motor neuron

bit.bio further expands ioCells™️ range with early access launch of ioMotor Neurons™️ for research and drug discovery in diseases such as amyotrophic lateral sclerosis (ALS)

Retrieved on: 
Wednesday, December 6, 2023
Health, Stem Cells, Genetics, Research, Science, Pharmaceutical, Biotechnology, CNS, ALS, Motor neuron diseases, Toxicology, Patient, Multimedia, Culture, Weakness, Automation, Motor neuron, Drug discovery, Neuron, Cell, CRISPR, Death, Brain cell, Innovation, Aroma compound, Pharmaceutical industry, Medical device

This takes the total number of products in the ioCells range - human cells for research and drug discovery - to 20 products, including wild type, CRISPR-ready and disease model cells.

Key Points: 
  • This takes the total number of products in the ioCells range - human cells for research and drug discovery - to 20 products, including wild type, CRISPR-ready and disease model cells.
  • The launch of ioMotor Neurons also follows the recent opening of bit.bio’s new manufacturing and automation laboratories by the UK’s Minister for Science, Research and Innovation.
  • Damage or death of motor neurons can lead to a devastating group of conditions called Motor Neuron Diseases (MNDs).
  • ioMotor Neurons are an important part of our ALS toolkit that can now be incorporated into motor neuron disease research and early drug discovery workflows in ways that were not previously possible.

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”.

Muscular Dystrophy Association Awards Venture Philanthropy Funding to PathMaker Neurosystems to Develop Neuromodulation Treatment for ALS

Retrieved on: 
Tuesday, January 17, 2023
Venture philanthropy, Neurodegeneration, Entrepreneurship, MVP, Muscular Dystrophy Association, Doctor of Philosophy, Biomedical engineering, MDA, Motor neuron, Amyotrophic lateral sclerosis, Degenerative disease, Muscular dystrophy, CERF, EFS, ALS, NIH, Pharmaceutical industry

BOSTON and NEW YORK, Jan. 17, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) and PathMaker Neurosystems Inc. (“PathMaker”), a near-commercial stage neuromodulation company developing breakthrough non-invasive systems for the treatment of serious neurological disorders, announced today an investment of $600,000 through the MDA Venture Philanthropy (MVP) program to PathMaker to advance its work in amyotrophic lateral sclerosis (ALS).

Key Points: 
  • BOSTON and NEW YORK, Jan. 17, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) and PathMaker Neurosystems Inc. (“PathMaker”), a near-commercial stage neuromodulation company developing breakthrough non-invasive systems for the treatment of serious neurological disorders, announced today an investment of $600,000 through the MDA Venture Philanthropy (MVP) program to PathMaker to advance its work in amyotrophic lateral sclerosis (ALS).
  • MDA uses the Venture Philanthropy program to invest in companies committed to clinical application of high-impact, novel treatments for neuromuscular diseases, including ALS.
  • This funding will support the launch of an ALS early feasibility study (EFS) using PathMaker’s breakthrough technology for motor neuron hyperexcitability suppression.
  • To learn more about the Muscular Dystrophy Association and the MVP program, please visit https://www.mda.org/science/mda-venture-philanthropy .

Stand Up Therapeutics Signed with VectorBuilder for World's First Gene Therapy Supply for Paraplegia Patient

Retrieved on: 
Thursday, December 29, 2022
Motor neuron, Fibroblast, Paralysis, Patient, Brain, Injury, Health, Spinal stenosis, Stroke, Spinal cord, University, Therapy, Myocardial infarction, Stand Up, Parkinson's disease, GMP, Paraplegia, SCI, Vaccine, Medical device

SEOUL, South Korea, Dec. 29, 2022 /PRNewswire/ -- VectorBuilder is the world's largest supplier of customized vectors for viral and non-viral gene delivery, operating as a multinational corporation headquartered in Chicago, USA with offices in North America, Europe, China, Japan, Korea, Australia and Israel. Currently, more than 80,000 custom vectors are produced annually for numerous researchers around the world. VectorBuilder has GMP facilities for gene delivery systems, and supplies gene therapy products and delivery systems for thousands of institutions around the world and more than 50,000 customers, including all the leading pharmaceutical companies and major universities. We are supplying gene therapy products and delivery systems.

Key Points: 
  • Stand Up Therapeutics Co., Ltd, located in South Korea, is a gene therapy company for paralyzed patients with spinal cord injury.
  • Stand Up Therapeutics announced that it had signed an agreement with VectorBuilder Inc., a global leader in gene delivery solutions, to manufacture a GMP grade gene delivery system.
  • According to Stand Up Therapeutics, through this agreement, VectorBuilder will produce and supply gene therapy products designed by Stand Up Therapeutics in the future.
  • VectorBuilder's Chief Scientist Bruce Lahn said, "VectorBuilder is very interested in Stand Up Therapeutics's gene therapy for the treatment of paralysis at the global level.

Pasithea Therapeutics to Present at Annual Meeting of the Antibody Society Conference

Retrieved on: 
Tuesday, December 6, 2022
Conference, Psychopharmacology, Sporadic group, Life expectancy, Multiple sclerosis, Behavior, Diagnosis, Noonan syndrome, Motor neuron, CNS, Chemistry, Heredity, National academy, Amyotrophy, ALS, FDA, Biotechnology, Death, Amyotrophic lateral sclerosis, Central nervous system, Spinal cord, Antibody, MIAMI, Alpha-5 beta-1, Neuroscience, Survival, KTTA, SOD1, Research, Neurodegeneration, Therapy, Integrin, Brain, Patient, Pharmaceutical industry

Pasithea’s discovery candidate, PAS-003, has a novel mechanism of action with the potential to improve clinical outcomes in patients with ALS.

Key Points: 
  • Pasithea’s discovery candidate, PAS-003, has a novel mechanism of action with the potential to improve clinical outcomes in patients with ALS.
  • “We’re pleased to present our data at such a prestigious annual conference that includes a keynote address by this year’s Nobel Laureate for chemistry,” commented Dr. Steinman.
  • Pasithea Therapeutics is a biotechnology company primarily focused on the discovery, research and development of innovative treatments for central nervous system (CNS) disorders.
  • PAS-003 is monoclonal antibody targeting alpha5/beta1 integrin for the treatment of Amyotrophic Lateral Sclerosis (ALS) and other neurological diseases.

Eikonoklastes Therapeutics and Forge Biologics Announce Viral Vector Contract Development and AAV Manufacturing Partnership

Retrieved on: 
Tuesday, November 29, 2022
Science, Neurology, Biotechnology, Research, Pharmaceutical, Health, Genetics, Clinical Trials, Ignition, Environment, Eikonoklastes, HEK 293, DNA, Multimedia, Therapy, Brain, Partnership, Patient, Movement, CGMP, Motor neuron, Triple-negative breast cancer, Paralysis, Neurodegeneration, ALS, Research, Spinal cord, Clinical trial, AAV, Amyotrophic lateral sclerosis, Forge, Neuron, Pharmaceutical industry, Vaccine, Hearth

View the full release here: https://www.businesswire.com/news/home/20221129005440/en/

Key Points: 
  • View the full release here: https://www.businesswire.com/news/home/20221129005440/en/
    Forge will provide adeno-associated viral (AAV) process development and manufacturing services for ET-101.
  • All development and AAV manufacturing activities will occur at the Hearth, Forges 200,000 square foot gene therapy facility in Columbus, Ohio.
  • Partnering our AAV manufacturing with Forge will further our ability to advance into clinical trials, said Bruce Halpryn, Ph.D., Chair, CEO, and Co-Founder of Eikonoklastes Therapeutics.
  • Forge Biologics is a hybrid gene therapy contract manufacturing and clinical-stage therapeutics development company.

PerkinElmer Announces its EONIS SCID-SMA Kit is First to Receive Marketing Authorization by U.S. FDA for SMA Screening in Newborns

Retrieved on: 
Monday, November 14, 2022
FDA, Other Health, Pharmaceutical, General Health, Medical Devices, Parenting, Children, Baby, Maternity, Family, Science, Biotechnology, Consumer, Other Science, Health, Research, Automation, FDA, Brainstem, Spinal muscular atrophy, Marketing, SCID, ESG, Food, Accreditation, Laboratory, Government, PerkinElmer, CE, PCR, DNA, Reproductive health, NYSE, Science, Motor neuron, SMA, Infant, Spinal cord, Software, Safety, Fundamental Analysis Software, Informatics, IVD, Birth, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, PKI, Degenerative disease, Inherited disorders of trafficking, Muscle weakness, Atrophy, Pharmaceutical industry

This is the first FDA authorized assay for SMA screening in newborns in the United States and is part of the Companys broader EONIS Platform .

Key Points: 
  • This is the first FDA authorized assay for SMA screening in newborns in the United States and is part of the Companys broader EONIS Platform .
  • This authorization is a major milestone for newborn screening in the United States.
  • Other components of the platform include the EONIS DNA Extraction kit and EONIS Analysis Software.
  • As the global leader in newborn screening, PerkinElmer offers solutions to help identify more than 50 congenital disorders.

Stevens Nation Responds to FDA's Refusal to File Letter, NurOwn Recipients Call for Advisory Committee Meeting

Retrieved on: 
Tuesday, November 22, 2022
Authorization, Muscle weakness, CDER, Brainstorm, Therapy, Camping, Atrophy, Eating, Refusal, FDA, Amyotrophic lateral sclerosis, Pulmonology, EAPS, Spinal cord, University of California, Irvine, Expanded access, RTF, UC, Advisory Committee, CDC, Patient, Phase, Aerospace, Walking, Motor neuron, Neurology, Brain, Life expectancy, Letter, Food, Exercise, Disease, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, EAP, Pharmaceutical industry, Medical device, Renewable energy, ALS

WASHINGTON, Nov. 21, 2022 /PRNewswire/ -- Brainstorm Cell Therapeutics is seeking FDA approval of its mesenchymal stem cell therapy called NurOwn for the treatment of ALS. In response to the FDA's Refusal to File letter, and as people who participated in the NurOwn trials and its Expanded Access program ("EAP"), we support Brainstorm's efforts to seek an Advisory Committee ("AdCom") meeting.

Key Points: 
  • In response to the FDA's Refusal to File letter, and as people who participated in the NurOwn trials and its Expanded Access program ("EAP"), we support Brainstorm's efforts to seek an Advisory Committee ("AdCom") meeting.
  • We must insist that the FDA allow testimony about the "clinically meaningful" changes that occurred when we received NurOwn.
  • However, the Phase III trial did meet a pre-specified secondary endpoint in a subgroup of patients with less advanced ALS.
  • The best people to attest to how NurOwn made them "feel and function" are the people who received NurOwn.

Eikonoklastes Therapeutics Receives FDA Orphan Drug Designation for 1st-in-class Treatment of Amyotrophic Lateral Sclerosis (ALS)

Retrieved on: 
Wednesday, November 9, 2022
FDA, Pharmaceutical, Health, Food, Movement, Neuron, Environment, University of California, San Diego Performance-Based Skills Assessment, FDA, Patient, Triple-negative breast cancer, ALS, Brain, Mortality, Survival, ODD, Clinical trial, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, University, Disease, Degenerative disease, Spinal cord, Eikonoklastes, Quality of life, Therapy, Paralysis, Motor neuron, Technology, CEO, Pharmaceutical industry

Eikonoklastes Therapeutics, a preclinical biotech company developing a 1st-in-class, AAV9-based gene therapy for treatment of sporadic and familial amyotrophic lateral sclerosis (ALS), announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for the ET-101 program.

Key Points: 
  • Eikonoklastes Therapeutics, a preclinical biotech company developing a 1st-in-class, AAV9-based gene therapy for treatment of sporadic and familial amyotrophic lateral sclerosis (ALS), announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for the ET-101 program.
  • The FDAs Orphan Drug Designation for ET-101 reflects the compelling data underlying the ET-101 program and its potential as a 1st-in-class treatment option for both familial and sporadic ALS, said Bruce Halpryn, chairman and CEO of Eikonoklastes Therapeutics.
  • Achieving Orphan Drug Designation is an important regulatory milestone that further validates our efforts to efficiently develop ET-101.
  • Eikonoklastes Therapeutics is a preclinical biotech company rapidly developing a pipeline of disruptive new treatments for todays most challenging diseases.

COUR Pharmaceuticals Announces FDA Clearance of Investigational New Drug Application for Myasthenia Gravis

Retrieved on: 
Tuesday, October 25, 2022
National Institute, MG, National Institute of Neurological Disorders and Stroke, Yale University, Muscle, Immunodeficiency, Communication, Disease, Assistant, IND, Autoimmune disease, Degenerative disease, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Lists of diseases, Patient, Research, Investigational New Drug, Takeda Pharmaceutical Company, Founder CEO, Nanoparticle, Muscle contraction, Motor neuron, Myasthenia gravis, Technology, Food, Immune system, Immune tolerance, Neurology, CEO, Face, Safety, FDA, Autoimmunity, Stroke, Myasthenia, Program, Vaccine, Pharmaceutical industry

The therapy is designed to treat the underlying autoimmune causes of myasthenia gravis (MG) through antigen-specific tolerance induced by CNP-106.

Key Points: 
  • The therapy is designed to treat the underlying autoimmune causes of myasthenia gravis (MG) through antigen-specific tolerance induced by CNP-106.
  • "CNP-106 has the potential to halt and reverse MG by reprogramming T cells to promote immune tolerance through T regulatory cells.
  • For the first time, we have an opportunity to address the likely seminal trigger which leads to neuromuscular junction damage and dysfunction."
  • "The FDA's acceptance of our IND application in myasthenia gravis represents COUR's fourth IND clearance to date and expands our focus deeper into rare and severe autoimmune diseases," said John J. Puisis, Founder & CEO of COUR.