SOD1

ProMIS Neurosciences Announces First Quarter 2024 Financial Results and Recent Highlights

Retrieved on: 
Dienstag, Mai 14, 2024
PROMIS, Patient, CSF, Pharmacokinetics, SOD1, Toxicity, Immunoglobulin G, Synucleinopathy, Trial of the century, Safety, ALS, Biochemistry, Blood, PMN, Bioorganic chemistry, Vaccination, BioRxiv, Cerebrospinal fluid, Journal, Multiple system atrophy, Research, Therapy, MSA, Biotechnology, Epitope, Neuroscience, Pharmaceutical industry

In January 2024, ProMIS announced the selection of a lead vaccine candidate, PMN400, against multiple synucleinopathies including MSA, Parkinson’s disease and Lewy Body Dementia.

Key Points: 
  • In January 2024, ProMIS announced the selection of a lead vaccine candidate, PMN400, against multiple synucleinopathies including MSA, Parkinson’s disease and Lewy Body Dementia.
  • In January 2024, ProMIS announced the appointment of Neil Warma as the interim Chief Executive Officer.
  • General and administrative expenses modestly increased to $1.6 million for the quarter ended March 31, 2024, compared to $1.4 million for the same period in 2023.
  • Net loss was $3.6 million for the quarter ended March 31, 2024, compared to a net loss of $5.0 million for the same period in 2023.

Voyager Therapeutics Presents Data for Second-Generation, TRACER™-Generated Capsids and CNS Gene Therapy Programs Advancing Toward Clinical Trials at the ASGCT 27th Annual Meeting

Retrieved on: 
Mittwoch, Mai 8, 2024
TRACER, Brain, Traffic barrier, Cell, Mouse, Neuron, Tropism, Machine learning, RNA, B cell, AAV, Voyager, IND, CNS, Alkaline phosphatase, Crab-eating macaque, Human, DNA, Therapy, Astrocyte, Gene, Society, ALS, Spinal cord, BBB, DSM-IV codes, Central nervous system, Messenger, Capsid, Transcytosis, NHP, ALPL, HEK293, Centrifuge, SOD1, Vaccine

LEXINGTON, Mass., May 08, 2024 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today announced the presentation of data related to its TRACER™ capsid discovery platform and TRACER-driven gene therapy programs at the American Society of Gene & Cell Therapy’s (ASGCT) 27th annual meeting.

Key Points: 
  • Voyager identified a highly conserved cell surface receptor that mediates enhanced brain tropism of the VCAP-101/102 engineered capsid class.
  • Voyager has advanced machine learning to predict production fitness of capsid variants with high accuracy, helping guide development of high-production-fit libraries.
  • Robust manufacturing processes are needed to remove empty and partially filled capsids from recombinant AAV (rAAV) drug product.
  • This cost-efficient cell line provides an AAV expression platform to produce drug candidates for neurological disorders.

uniQure Announces First Quarter 2024 Financial Results and Highlights Recent Company Progress

Retrieved on: 
Dienstag, Mai 7, 2024
FDA, Phase, Safety, Food, Fabry disease, Research, Immunosuppression, Male, Hand, IRB, Conference, Information technology, RBC Capital Markets, Patient, ALS, Acquisition, CSL, POC, Clinical trial, Temporal lobe epilepsy, SOD1, Pharmaceutical industry

LEXINGTON, Mass. and AMSTERDAM, May 07, 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 first quarter of 2024 and highlighted recent progress across its business.

Key Points: 
  • ~ Clinical trial initiation for Fabry disease on track to begin in second quarter of 2024, followed by refractory mesial temporal lobe epilepsy and SOD1-ALS in third quarter of 2024 ~
    LEXINGTON, Mass.
  • and AMSTERDAM, May 07, 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 first quarter of 2024 and highlighted recent progress across its business.
  • We have obtained IRB approvals for initial sites, which are poised for imminent activation, and have also identified prospective patients.
  • The Company expects cash, cash equivalents and investment securities will fund operations into the second quarter of 2027.

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

Retrieved on: 
Montag, April 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

Retrieved on: 
Dienstag, April 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

Retrieved on: 
Dienstag, April 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

Retrieved on: 
Mittwoch, März 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

Retrieved on: 
Mittwoch, Februar 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

Retrieved on: 
Mittwoch, Februar 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: 
Montag, Februar 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.