SOD1

Voyager Therapeutics to Present Broad Set of Translational Data Supporting IV-Delivered, CNS Gene Therapy Programs Advancing Toward Clinical Trials at the ASGCT 27th Annual Meeting

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星期一, 四月 22, 2024
TRACER, Histology, Mouse, Phenotype, Data science, Cell, AAV, CNS, Machine learning, Senior, Tauopathy, Gene, Process, Society, BBB, ALS, Central nervous system, Public, HEK293, Therapy, Centrifuge, SOD1, Vaccine

“Voyager’s novel TRACER-derived capsids underlie 13 partnered programs and three wholly-owned programs to enable IV-delivery of gene therapies for diseases of the central nervous system.

Key Points: 
  • “Voyager’s novel TRACER-derived capsids underlie 13 partnered programs and three wholly-owned programs to enable IV-delivery of gene therapies for diseases of the central nervous system.
  • Three of those programs now have development candidates selected, and we see the potential for them to enter clinical trials next year,” said Todd Carter, Ph.D., Chief Scientific Officer of Voyager Therapeutics.
  • ET
    Intravenous administration of BBB-penetrant, MAPT-Silencing, AAV gene therapy provides broad and robust CNS Tau lowering in tauopathy mouse models (#1602).
  • ET
    Intravenous delivery of AAV gene therapy for the treatment of SOD1-ALS provides broad SOD1 lowering in NHP (#1647).

Voyager Therapeutics Announces Selection of Development Candidate for GBA1 Program in Collaboration with Neurocrine Biosciences, Triggering Milestone Payment

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星期二, 四月 16, 2024
FDA, TRACER, Neurocrine Biosciences, Traffic barrier, Neurocrine, Research, Sympathetic nervous system, AAV, IND, GBA1, DSM-IV codes, FA, Clinical trial, Therapy, Investigational New Drug, SOD1, Pharmaceutical industry

The candidate combines a GBA1 gene replacement payload with an intravenously administered, blood-brain barrier penetrant, novel capsid derived from Voyager’s TRACER™ capsid discovery platform.

Key Points: 
  • The candidate combines a GBA1 gene replacement payload with an intravenously administered, blood-brain barrier penetrant, novel capsid derived from Voyager’s TRACER™ capsid discovery platform.
  • Selection of the development candidate triggered a $3 million milestone payment to Voyager, which the Company expects to receive in the second quarter of 2024.
  • Voyager is eligible to receive additional future development and commercialization milestone payments based on the further advancement of this program.
  • “The nomination of this development candidate in the GBA1 program, following the recent nomination of a development candidate in the Friedreich’s ataxia program, demonstrates the productivity of the collaboration between Voyager and Neurocrine to advance gene therapies for neurological diseases,” said Alfred W. Sandrock, Jr., M.D., Ph.D., Chief Executive Officer of Voyager.

ProMIS Neurosciences Publishes in the Journal of Biological Chemistry on the Interaction Between Pathogenic Proteins as a Treatment Target for ALS

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星期二, 四月 9, 2024
Toxicity, PMN, MSA, Bioorganic chemistry, ALS, Multiple system atrophy, Zebrafish, SOD1, Neuroscience, Journal, Brain cell, TAR, Protein, Biochemistry, PROMIS, Antibody, Therapy, Vaccine

ProMIS is developing antibodies selectively targeting misfolded forms of TDP-43 and SOD1.

Key Points: 
  • ProMIS is developing antibodies selectively targeting misfolded forms of TDP-43 and SOD1.
  • ALS is a fatal neurodegenerative disease of motor neurons.
  • “Publication of these data underscores the connection of misfolded proteins and ALS and supports targeting our TDP-43-specific epitope with PMN267 as a potential therapeutic approach,” stated Neil Warma, Chief Executive Officer of ProMIS Neurosciences.
  • “PMN267 is advancing through preclinical development and is showing promise as a potential treatment for ALS.

Voyager Therapeutics Announces Appointment of Toby Ferguson as Chief Medical Officer

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星期三, 三月 13, 2024
AAV, ALS, Assistant, Central nervous system, University, Florida College, SOD1, CNS, Running, Traffic barrier, IND, Biogen, DSM-IV codes, Therapy, CMO, Medical director, University of Florida, Pharmaceutical industry

LEXINGTON, Mass., March 13, 2024 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today announced the appointment of Toby Ferguson, M.D., Ph.D., as Chief Medical Officer (CMO), effective March 25, 2024.

Key Points: 
  • LEXINGTON, Mass., March 13, 2024 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today announced the appointment of Toby Ferguson, M.D., Ph.D., as Chief Medical Officer (CMO), effective March 25, 2024.
  • During his tenure, he built and developed teams focused on neuromuscular and movement disorders, overseeing strategy for these areas across Biogen R&D.
  • Prior to joining Biogen, Ferguson was Assistant Professor of Neurology, Shriners Pediatric Research Center and Temple University School of Medicine.
  • Voyager represents for me an extraordinary opportunity to fulfill this mission.”

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

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星期三, 二月 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.

uniQure Announces 2023 Financial Results and Highlights Recent Company Progress

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星期三, 二月 28, 2024
Acquisition, Fabry disease, TMS, Natural history, CSL Behring, ALS, Research, FDA, Partnership, Cerebrospinal fluid, Food, CSL, Etranacogene dezaparvovec, Sale, TFC, Conference, SOD1, Unified, Information technology, AFL, Immunosuppression, Patient, Disease, Phase, Hand, IND, CGMP, State, Temporal lobe epilepsy, Pharmaceutical industry

LEXINGTON, Mass. and AMSTERDAM, Feb. 28, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the fourth quarter and full year 2023 and highlighted recent progress across its business.

Key Points: 
  • and AMSTERDAM, Feb. 28, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the fourth quarter and full year 2023 and highlighted recent progress across its business.
  • “We are pleased with the progress made across the company in 2023 and are now laser-focused on execution across multiple clinical programs,” stated Matt Kapusta, chief executive officer of uniQure .
  • By the end of 2024, the Company expects to have greater clarity regarding a potential approval pathway for AMT-130.
  • AMT-191 for the treatment of Fabry disease – In November 2023, the Company announced the clearance of an IND for the Phase I/IIa clinical study of AMT-191.

Voyager Therapeutics Announces Selection of Gene Therapy Development Candidate for Friedreich’s Ataxia in Collaboration with Neurocrine Biosciences, Triggering Milestone Payment

Retrieved on: 
星期一, 二月 26, 2024
Traffic barrier, ALS, SOD1, Neurocrine Biosciences, Therapy, Neurocrine, FXN, GBA1, AAV, Research, FA, Vaccine

The candidate combines a frataxin (FXN) gene replacement payload with an intravenously administered, blood-brain barrier penetrant, novel capsid derived from Voyager’s TRACER™ capsid discovery platform.

Key Points: 
  • The candidate combines a frataxin (FXN) gene replacement payload with an intravenously administered, blood-brain barrier penetrant, novel capsid derived from Voyager’s TRACER™ capsid discovery platform.
  • Selection of the development candidate triggered a $5 million milestone payment to Voyager, which the Company expects to receive in the first quarter of 2024.
  • Voyager is eligible to receive additional future development and commercialization milestone payments based on the further advancement of this program.
  • We believe our strategy to replace the defective frataxin gene could address the underlying disease etiology of FA.

Biogen’s QALSODY® (tofersen), the First Therapy to Treat Rare, Genetic Form of ALS, Received Positive Opinion from CHMP

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星期五, 二月 23, 2024
Fever, European Commission, EMA, ALS, Committee, MND, Myalgia, Intracranial pressure, BIIB, Cerebrospinal fluid, Pain, Hope, Biogen, European, SOD1, Arthralgia, Civil service commission, Biomarker, Fatigue, CHMP, AFL, European Medicines Agency, Protein, Disease, UZ Leuven, Mutation, Myelitis, Neurology, Aseptic meningitis, Research, Chronic pain, Vaccine

If authorized by the European Commission (EC), QALSODY will be the first treatment approved in the European Union to target a genetic cause of ALS, also known as motor neuron disease (MND).

Key Points: 
  • If authorized by the European Commission (EC), QALSODY will be the first treatment approved in the European Union to target a genetic cause of ALS, also known as motor neuron disease (MND).
  • “The CHMP’s positive opinion reinforces the impact QALSODY can have in SOD1-ALS and further demonstrates Biogen’s commitment to address the unmet needs of people living with ALS and neuromuscular diseases,” said Priya Singhal, M.D., M.P.H., Head of Development at Biogen.
  • Trends towards improvement in the physical abilities of participants who received QALSODY were seen compared to those who received placebo, as measured by the ALS Functional Ratings Scale-Revised (ALSFRS-R).
  • Serious neurologic events, including myelitis and/or radiculitis; papilledema and elevated intracranial pressure; and aseptic meningitis have also been reported.

Gain Therapeutics’ GT-02287 Completely Restores Motor Function in Mouse Models of Parkinson’s Disease

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星期二, 二月 6, 2024
Neuroprotection, Poster, Pharmacokinetics, ALS, AFL, Clinical trial, Patient, Safety, Mutation, FDA, Animal, Food, MPS II, Disease, Therapy, MPS, Neurofilament, SOD1, Glucocerebrosidase, GBA1, Plasma, Pharmaceutical industry

The data was accepted as a late-breaker abstract and will be presented at the 20th Annual WORLDSymposium™ being held in San Diego this week.

Key Points: 
  • The data was accepted as a late-breaker abstract and will be presented at the 20th Annual WORLDSymposium™ being held in San Diego this week.
  • “We believe the data showing complete restoration of motor function in a therapeutic model are remarkable and further support the potential of GT-02287 to slow or stop the progression of Parkinson’s disease, a disease for which only symptomatic treatments are available to patients at this time,” said Matthias Alder, Gain Therapeutics’ Chief Executive Officer.
  • Further, animals in the most challenging treatment group – those that began treatment eight days following onset of the disease – showed motor improvement from day 14 to day 27, which suggests progressive reversal of neuronal deficit associated with continued treatment duration.
  • Further details of the study, including protocol and specific results can be found in the poster, which was presented today and can be accessed here .

Orphan designation: Adeno-associated viral vector serotype rh10 encoding miRNA against SOD1 mRNA Treatment of amyotrophic lateral sclerosis, 08/11/2023 Positive

Retrieved on: 
星期日, 二月 4, 2024
ALS, COMP, IRIS, EMA, European Commission, Messenger, Patient, SOD1, MicroRNA, Committee, Pharmaceutical industry

Key facts

Key Points: 
  • Key facts
    - Active substance
    - Adeno-associated viral vector serotype rh10 encoding miRNA against SOD1 mRNA
    - Intended use
    - Treatment of amyotrophic lateral sclerosis
    - Orphan designation status
    - Positive
    - EU designation number
    - EU/3/23/2859
    - Date of designation
    - Sponsor
    uniQure biopharma B.V.
  • Patients' organisations
    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
    European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:
    EMA list of opinions on orphan medicinal product designation
    EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform: