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Details:
LYS-SAF302 (olenasufligene relduparvovec) is a novel gene therapy in Phase 2/3 development for the treatment of MPS IIIA, or Sanfilippo syndrome type A. LYS-SAF302 delivers a functional copy of the human SGSH gene directly to brain cells using the AAV carrier.
Lead Product(s): Olenasufligene Relduparvovec
Therapeutic Area: Genetic Disease Product Name: LYS-SAF302
Highest Development Status: Phase II/ Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 18, 2022
Details:
LYS-SAF302 (olenasufligene relduparvovec) delivers a working copy of the human SGSH gene directly to the brain through intra-cerebral injection. Mutations to the SGSH gene are responsible for the developmental, physical and behavioral symptoms of the disease.
Lead Product(s): Olenasufligene Relduparvovec
Therapeutic Area: Genetic Disease Product Name: LYS-SAF302
Highest Development Status: Phase II/ Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 18, 2022
Details:
Preliminary results evaluating LYS-SAF302 (olenasufligene relduparvovec) in patients with Sanfilippo syndrome type A who have been followed for at least two years demonstrated improvement, stabilization, or slowing down of decline in cognitive-developmental age.
Lead Product(s): Olenasufligene Relduparvovec
Therapeutic Area: Genetic Disease Product Name: LYS-SAF302
Highest Development Status: Phase II/ Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 18, 2022
Details:
AAVance trial evaluates, effectiveness of a one-time intracerebral delivery of a recombinant adeno-associated virus vector rh.10 carrying the N-sulfoglucosamine sulfohydrolase gene LYS-SAF302 (olenasufligene relduparvovec) in children with MPS IIIA.
Lead Product(s): Olenasufligene Relduparvovec
Therapeutic Area: Genetic Disease Product Name: LYS-SAF302
Highest Development Status: Phase II/ Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 24, 2022
Details:
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.
Lead Product(s): Olenasufligene Relduparvovec
Therapeutic Area: Genetic Disease Product Name: LYS-SAF302
Highest Development Status: Phase II/ Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Lysogene
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Termination January 13, 2022
Details:
Following discussions with the U.S. FDA, a clinical hold was issued for the clinical trial AAVance, a global Phase 2/3 clinical trial of LYS-SAF302, a gene therapy for the treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA, also known as Sanfilippo syndrome type A).
Lead Product(s): Olenasufligene Relduparvovec
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase II/ Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 05, 2020
Details:
The AAVance Phase 2/3 clinical study is designed as an open-label, single-arm, multicenter study of LYS-SAF302 for the treatment of MPS IIIA.
Lead Product(s): Olenasufligene Relduparvovec
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase II/ Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 25, 2020
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