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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:
The publication of a peer-reviewed article for FXS01 in EMBO Molecular Medicine showing that adeno-associated viral (AAV) vector-delivered diacylglycerol kinase (DGKk) achieves long-term rescue of fragile X syndrome (FXS) in a mouse model of disease.
Lead Product(s): FXS01
Therapeutic Area: Genetic Disease Product Name: FXS01
Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable April 21, 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:
Lysogene has dosed the first patient in France with LYS-GM101 investigational gene therapy at the Armand Trousseau Hospital (AP-HP). This is the third patient treated with LYS-GM101 in the global adaptative-design clinical trial in children with GM1 gangliosidosis.
Lead Product(s): AAVrh.10 Expressing Beta-galactosidase
Therapeutic Area: Genetic Disease Product Name: LYS-GM101
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 07, 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
Recipient: Sarepta Therapeutics
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Termination January 13, 2022
Details:
LYS-GM101 investigational gene therapy an ‘Innovation Passport’ for the treatment of GM1 gangliosidosis under the United Kingdom’s (UK) Innovative Licensing and Access Pathway (ILAP). LYS-GM101 is currently in clinical development.
Lead Product(s): AAVrh.10 Expressing beta-galactosidase
Therapeutic Area: Genetic Disease Product Name: LYS-GM101
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 20, 2021
Details:
This financing will support the clinical study of LYS-GM101 drug candidate for the treatment of GM1 gangliosidosis and the preclinical studies of LYS-FXS01 in Fragile X syndrome.
Lead Product(s): LYS-GM101
Therapeutic Area: Genetic Disease Product Name: LYS-GM101
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Bpifrance
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Financing November 26, 2021
Details:
LYS-GM101 (‘adeno-associated viral vector serotype rh.10 expressing beta-galactosidase’) received orphan drug designation for the treatment of GM1 gangliosidosis in the European Union and in the US in 2017, as well as the Rare Pediatric Disease designation in the US in 2016.
Lead Product(s): AAVrh.10 Expressing Beta-galactosidase
Therapeutic Area: Genetic Disease Product Name: LYS-GM101
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 31, 2021
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