Lysogenic cycle

Aura Biosciences Strengthens and Expands Leadership Team with Key Appointments

Retrieved on: 
Monday, October 2, 2023
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“Jill and Mark’s appointments come at an important time in Aura’s evolution as a late stage clinical development company,” said Elisabet de los Pinos, Ph.D., Chief Executive Officer of Aura.

Key Points: 
  • “Jill and Mark’s appointments come at an important time in Aura’s evolution as a late stage clinical development company,” said Elisabet de los Pinos, Ph.D., Chief Executive Officer of Aura.
  • “We have built our leadership team with experienced industry leaders who have proven track records of success with multiple drug approvals.
  • Dr. Hopkins is board certified in Ophthalmology from the American Board of Ophthalmology and the Royal College of Surgeons Canada.
  • Before joining Sanofi Genzyme, Dr. Plavsic held various technical leadership positions with AstraZeneca, Q-One Biotech, and Life Technologies.

Sarepta Therapeutics Announces Fourth Quarter and Full-Year 2022 Financial Results and Recent Corporate Developments

Retrieved on: 
Tuesday, February 28, 2023
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Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the fourth quarter and full-year 2022.

Key Points: 
  • Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the fourth quarter and full-year 2022.
  • VOYAGENE is a phase 1 study of SRP-9003 (bidridistrogene xeboparvovec) for the treatment of limb-girdle muscular dystrophy Type 2E (LGMD2E).
  • These non-GAAP measures are not intended to be considered in isolation or to replace the presentation of the Company’s financial results in accordance with GAAP.
  • All relevant non-GAAP measures are reconciled from their respective GAAP measures in the attached table "Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures.”

Lysogene Provides Additional Update on AAVance Phase 2/3 Gene Therapy Clinical Trial with LYS-SAF302 in children with MPS IIIA

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Thursday, July 7, 2022
Health, Genetics, Science, Research, Clinical Trials, NFL, Nerve, Mutation, Academic Model Providing Access to Healthcare (AMPATH), Patient, Lists of diseases, HS, Cerebrospinal fluid, Tissue, Brain, CHOC, Phenotype, Adaptive clinical trial, AAV, CNS, CSF, Central nervous system, Company, Spinal cord, Disease, MPS, Autism, SGSH, DA, GM1, Weizmann Institute of Science, Serum, Infant, Clinical trial, Parkinson, Yeda, Child, CA, LYS, Parkinson's disease, Foundation, SD, Fragile X syndrome, Language, Database, Competition and Markets Authority, GBA1, Oligosaccharide, Observation, Forward-looking statement, Conference, Sanfilippo, Medical device, Pharmaceutical industry, Lysogenic cycle

Lysogene (Paris:LYS)(FR0013233475 LYS), a phase 3 gene therapy platform Company targeting central nervous system (CNS) diseases, today communicates additional preliminary data from the AAVance Phase 2/3 gene therapy trial in MPS IIIA (NCT03612869).

Key Points: 
  • Lysogene (Paris:LYS)(FR0013233475 LYS), a phase 3 gene therapy platform Company targeting central nervous system (CNS) diseases, today communicates additional preliminary data from the AAVance Phase 2/3 gene therapy trial in MPS IIIA (NCT03612869).
  • Lysogene is a gene therapy Company focused on the treatment of orphan diseases of the central nervous system (CNS).
  • A phase 2/3 clinical trial in MPS IIIA is ongoing.
  • Lysogene is also developing an innovative AAV gene therapy approach for the treatment of Fragile X syndrome, a genetic disease related to autism.

Lysogene Provides Update on Efficacy, Safety and Timelines of the AAVance Phase 2/3 Clinical Trial with Gene Therapy LYS-SAF302 for the Treatment of MPSIIIA

Retrieved on: 
Thursday, February 24, 2022
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Lysogene (FR0013233475 LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today provides an update on clinical results and timelines of the ongoing AAVance phase 2/3 clinical trial with the investigational gene therapy LYS-SAF302 for the treatment of MPS IIIA (NCT03612869).

Key Points: 
  • Lysogene (FR0013233475 LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today provides an update on clinical results and timelines of the ongoing AAVance phase 2/3 clinical trial with the investigational gene therapy LYS-SAF302 for the treatment of MPS IIIA (NCT03612869).
  • LYS-SAF302 is intended to deliver a functional copy of the SGSH gene and allow the brain to secrete the missing enzyme.
  • In terms of safety, white matter abnormalities were observed near injection sites of patients treated in AAVance.
  • Lysogene is a gene therapy Company focused on the treatment of orphan diseases of the central nervous system (CNS).

Lysogene Reports Additional Positive Biomarker Data With LYS-SAF302 and Favorable Safety Data With LYS-GM101 Presented at the WORLDSymposium™ 2022

Retrieved on: 
Friday, February 11, 2022
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Data were presented in oral presentations at the WORLDSymposium 2022, on February 10th, 2022.

Key Points: 
  • Data were presented in oral presentations at the WORLDSymposium 2022, on February 10th, 2022.
  • Following treatment with LYS-SAF302 in AAVance, mean serum NF-L concentrations increased, reaching about 2-fold above baseline at 6 months.
  • We look forward to confirming these results in additional patients and timepoints, said Dr. Ralph Laufer, Chief Scientific Officer of Lysogene.
  • Lysogene is a gene therapy Company focused on the treatment of orphan diseases of the central nervous system (CNS).

 Lysogene Announces Termination of License Agreement With Sarepta for LYS-SAF302 Program, Regaining Global Commercial Rights

Retrieved on: 
Thursday, January 13, 2022
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This termination follows unsuccessful discussions for transferring back to Lysogene the responsibility for the global commercial supply of LYS-SAF302.

Key Points: 
  • This termination follows unsuccessful discussions for transferring back to Lysogene the responsibility for the global commercial supply of LYS-SAF302.
  • The termination of the agreement will enable Lysogene to regain development and commercialization rights for LYS-SAF302 in the US and other non-EU territories as well as the responsibility for global commercial supply of LYS-SAF302, all previously granted to Sarepta.
  • Lysogene will be entitled to receive reimbursement for certain costs associated with the termination.
  • Karen Aiach, Chairman and Chief Executive Officer of Lysogene commented: I wanted to thank Sarepta for helping us take the LYS-SAF302 program forward over the past years.

NanoView Biosciences Announces Key Appointments to Scientific Advisory Board

Retrieved on: 
Thursday, August 19, 2021
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BOSTON, Aug. 19, 2021 /PRNewswire/ --NanoView Biosciences today announced the appointment of Prof. Jan Ltvall, M.D., Ph.D., and Mark Plavsic, DVM, Ph.D., to its Scientific Advisory Board.

Key Points: 
  • BOSTON, Aug. 19, 2021 /PRNewswire/ --NanoView Biosciences today announced the appointment of Prof. Jan Ltvall, M.D., Ph.D., and Mark Plavsic, DVM, Ph.D., to its Scientific Advisory Board.
  • Jan Ltvall is a Professor at the University of Gothenburg in Sweden where he directs a research laboratory studying extracellular vesicles.
  • He is a medical specialist in both Clinical Allergy and Clinical Pharmacology, and has extensive experience in translational studies in inflammation and cancer.
  • I am excited to advise NanoView as it delivers unique products and services to advance this rapidly expanding field of nanomedicine," stated Prof. Ltvall.