Motor neuron disease

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

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Tuesday, January 31, 2023
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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.

Massachusetts General Hospital Reports Results of Interim Analysis from REFINE-ALS Biomarker Trial by Mitsubishi Tanabe Pharma USA, Utilizing Oxford Biodynamics’ EpiSwitch® Platform for Prognostic Stratification of Fast Progression ALS

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Friday, December 16, 2022
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The results from a subset of data, highlight the blood-based EpiSwitch prognostic stratification of patients with fast progressing Amyotrophic Lateral Sclerosis (ALS).

Key Points: 
  • The results from a subset of data, highlight the blood-based EpiSwitch prognostic stratification of patients with fast progressing Amyotrophic Lateral Sclerosis (ALS).
  • [7]
    Edaravone was discovered and developed for ALS by Mitsubishi Tanabe Pharma Corporation (MTPC) and Mitsubishi Tanabe Pharma Development America, Inc. (MTDA), commercialized in the U.S. by Mitsubishi Tanabe Pharma America, Inc. (MTPA).
  • Oxford BioDynamics notes first patient enrolled in ALS biomarker study sponsored by Mitsubishi Tanabe Pharma America, 25 Oct 2019. https://otp.tools.investis.com/clients/uk/oxford_biodynamics_plc/rns/reg...
    [5] Proceedings of the 21st Annual Meeting of NEALS Consortium.
  • Interim Analysis of the Radicava/Edaravone Findings in Biomarkers from ALS (REFINE-ALS) Study, Nov 2022. https://onlinelibrary.wiley.com/doi/epdf/10.1002/mus.27729
    [6] ALS News Today.

Emerging Pipeline Therapeutics Across 6 Rare Neuromuscular Diseases: Strategic Partnering and New Product Development will Enable Better Biologics for Personalized and Targeted Treatment - ResearchAndMarkets.com

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Tuesday, December 13, 2022
Biotechnology, Pharmaceutical, Health, Myasthenia gravis, Sarepta, Therapy, Spinal muscular atrophy, CIDP, DN, US FDA, DMD, Disorder, Myopathy, SMA, Duchenne muscular dystrophy, NMD, Golodirsen, Motor neuron disease, Patient, Amyotrophic lateral sclerosis, US, Degenerative disease, Research, Neuromuscular Disorders, NMDS, ALS, Diabetic neuropathy, Eteplirsen, Muscle, Cramp, Quality of life, Muscle weakness, RNA, Patent, Pharmaceutical industry, Vaccine

Biologics presents a personalized and targeted method to treat NMDs and is a promising treatment class.

Key Points: 
  • Biologics presents a personalized and targeted method to treat NMDs and is a promising treatment class.
  • This research, "Emerging Therapeutics for Rare Neuromuscular Diseases: A Pipeline Analysis" explores emerging biologics across these six NMDs.
  • Key biologics emerging for these rare NMDs include stem cell therapies, other cell-based treatments, gene therapies, RNA therapeutics, immunotherapies, and other protein/peptide-based treatments.
  • Therefore, biologics are suitably poised to transform the rare NMD landscape with personalized and targeted treatment strategies and will provide life-saving treatment options for fatal neuromuscular conditions.

Emerging Therapeutics for Rare Neuromuscular Diseases: Pipeline Analysis

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Thursday, December 1, 2022
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Muscle weakness, cramps, and impaired musculoskeletal functions that occur due to rare defects in nerve and muscle cells characterize neuromuscular disorders (NMDs).

Key Points: 
  • Muscle weakness, cramps, and impaired musculoskeletal functions that occur due to rare defects in nerve and muscle cells characterize neuromuscular disorders (NMDs).
  • The disorders are largely classified as motor neuron diseases, hereditary ataxias, peripheral nerve disorders, neuromuscular junction transmission disorders, and myopathies.
  • Frost & Sullivans research, Emerging Therapeutics for Rare Neuromuscular Diseases: A Pipeline Analysis explores emerging biologics across these six NMDs.Key biologics emerging for these rare NMDs include stem cell therapies, other cell-based treatments, gene therapies, RNA therapeutics, immunotherapies, and other protein/peptide-based treatments.
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NeuroSense Therapeutics to Present at Upcoming Leading Neuroscience Conferences

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Thursday, October 27, 2022
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CAMBRIDGE, Mass., Oct. 27, 2022 /PRNewswire/ -- NeuroSense Therapeutics Ltd. (NASDAQ: NRSN) ("NeuroSense"), a company developing treatments for severe neurodegenerative diseases, today announced its participation in three upcoming conferences.

Key Points: 
  • CAMBRIDGE, Mass., Oct. 27, 2022 /PRNewswire/ -- NeuroSense Therapeutics Ltd. (NASDAQ: NRSN) ("NeuroSense"), a company developing treatments for severe neurodegenerative diseases, today announced its participation in three upcoming conferences.
  • Dr. Shiran Zimri, NeuroSense's Head of Scientific Program, will present a poster on NeuroSense's recent biomarker research, developments, and findings.
  • Dr. Niva Russek-Blum, NeuroSense' VP Discovery & IP Generator, will also attend the conference, exploring potential scientific collaborations and partnership opportunities.
  • NeuroSense Therapeutics, Ltd. is a clinical-stage biotechnology company focused on discovering and developing treatments for patients suffering from debilitating neurodegenerative diseases.

EQS-News: New Drugs And Therapies Are Giving MS And ALS Sufferers New Hope

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Monday, October 10, 2022
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People suffering from incurable neurological diseases such as multiple sclerosis (MS) and amyotrophic lateral sclerosis (ALS) hope new therapies and drugs will arrive sooner than later.

Key Points: 
  • People suffering from incurable neurological diseases such as multiple sclerosis (MS) and amyotrophic lateral sclerosis (ALS) hope new therapies and drugs will arrive sooner than later.
  • MS is a potentially disabling disease affecting the brain and spinal cord the bodys central nervous system.
  • The immune system of those with MS attacks the myelin, the protective sheath covering a persons nerve fibers.
  • Signs and symptoms of MS vary widely and depend on the amount of nerve damage and which nerves are affected.

Stryker Launches New Gamma4 Hip Fracture Nailing System

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Thursday, September 1, 2022
Medical Supplies, Technology, Managed Care, Other Health, Health, Radiology, Health Technology, Medical Devices, Hospitals, Surgery, Hardware, Femur, NYSE, Trochanter, Olga Forsh, Mount Sinai Hospital, Spine, Consultant, Screw, Motor neuron disease, Fracture, Multimedia, SYK, Precision, Stryker Corporation, Surgeon, IMN, Feedback, Medical and Surgical History of the War of the Rebellion, Maxey, EPS, General manager, Patient, Surveying, Dentistry, DRS, Roc, Trauma, Osteoporosis

Stryker (NYSE:SYK) has launched the Gamma4 System, strengthening the products 30-year legacy of continuous innovation and clinical history.

Key Points: 
  • Stryker (NYSE:SYK) has launched the Gamma4 System, strengthening the products 30-year legacy of continuous innovation and clinical history.
  • The newest Gamma System will provide surgeons with the next generation of Strykers intramedullary nailing system.
  • View the full release here: https://www.businesswire.com/news/home/20220901005173/en/
    Stryker's Gamma System will provide surgeons with the next generation of Strykers intramedullary nailing system.
  • Being one of the most commonly used devices to repair a hip fracture, Im confident that we met our goal of reshaping patient hip fracture care for many years to come.

Pasithea Therapeutics Awarded a Drug Development Research Grant

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Thursday, July 14, 2022
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MIAMI BEACH, Fla., July 14, 2022 (GLOBE NEWSWIRE) -- Pasithea Therapeutics Corp. (Nasdaq: KTTA) Pasithea Therapeutics Corp. (Nasdaq: KTTA) (“Pasithea” or the “Company”), a novel biotechnology company focused on the research and discovery of new and effective treatments for psychiatric and neurological disorders, today announced the availability of an AUD $1 million (U.S. $694,000) drug development research award following the Company's acquisition of Alpha-5 integrin, LLC (“Alpha-5”). The grant was awarded by FightMND, the largest independent funder of amyotrophic lateral sclerosis (“ALS”)/motor neuron disease (“MND”) research in Australia.

Key Points: 
  • -- AUD $1 million (U.S $694,000) grant transferred as part of Alpha-5 Integrin, LLC transaction --
    MIAMI BEACH, Fla., July 14, 2022 (GLOBE NEWSWIRE) -- Pasithea Therapeutics Corp. (Nasdaq: KTTA) Pasithea Therapeutics Corp. (Nasdaq: KTTA) (Pasithea or the Company), a novel biotechnology company focused on the research and discovery of new and effective treatments for psychiatric and neurological disorders, today announced the availability of an AUD $1 million (U.S. $694,000) drug development research award following the Company's acquisition of Alpha-5 integrin, LLC (Alpha-5).
  • The grant was awarded by FightMND, the largest independent funder of amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND) research in Australia.
  • Ultimately, we aim to help ALS/MND patients who are in dire need of effective therapy, stated Dr. Tiago Reis Marques, CEO of Pasithea.
  • Pasithea Therapeutics Corporation is a U.S. biotechnology company focused on the research and discovery of new and effective treatments for psychiatric and neurological disorders.

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

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Wednesday, July 6, 2022
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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.

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.