Lead Product(s) : Olenasufligene Relduparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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 dis...
Product Name : LYS-SAF302
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
November 18, 2022
Lead Product(s) : Olenasufligene Relduparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II/ Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Olenasufligene Relduparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lysogene Provides Updates and Topline Results from Phase 2/3 AAVance Gene Therapy Clinical Study
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 ca...
Product Name : LYS-SAF302
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
November 18, 2022
Lead Product(s) : Olenasufligene Relduparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II/ Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Olenasufligene Relduparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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-developmen...
Product Name : LYS-SAF302
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
May 18, 2022
Lead Product(s) : Olenasufligene Relduparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II/ Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Olenasufligene Relduparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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.
Product Name : LYS-SAF302
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
February 24, 2022
Lead Product(s) : Olenasufligene Relduparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II/ Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Olenasufligene Relduparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Lysogene
Deal Size : Undisclosed
Deal Type : Termination
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.
Product Name : LYS-SAF302
Product Type : Cell and Gene therapy
Upfront Cash : Undisclosed
January 13, 2022
Lead Product(s) : Olenasufligene Relduparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II/ Phase III
Sponsor : Lysogene
Deal Size : Undisclosed
Deal Type : Termination
Lead Product(s) : Olenasufligene Relduparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : AAVance clinical trial is a global Phase 2/3 gene therapy trial for the treatment of Mucopolysaccharidosis. The company remains committed to the LYS-SAF302 development program and the Sanfilippo patient community.
Product Name : LYS-SAF302
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
October 26, 2020
Lead Product(s) : Olenasufligene Relduparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II/ Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Olenasufligene Relduparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Company has announced death of a patient in the AAVance study , a phase 2/3 international clinical study evaluating gene therapy treatment of Mucopolysaccharidosis type IIIA (MPS IIIA, known as Sanfilippo Syndrome type A) .
Product Name : LYS-SAF302
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
October 15, 2020
Lead Product(s) : Olenasufligene Relduparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II/ Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Olenasufligene Relduparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lysogene Announces Important Updates on AAVance Phase 2/3 Clinical Trial with LYS-SAF302 in MPS IIIA
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 s...
Product Name : LYS-SAF302
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
May 06, 2020
Lead Product(s) : Olenasufligene Relduparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II/ Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Olenasufligene Relduparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lysogene Receives FDA Fast Track Designation for LYS-SAF302 Gene Therapy in MPS IIIA
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.
Product Name : LYS-SAF302
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
February 25, 2020
Lead Product(s) : Olenasufligene Relduparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II/ Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable