

Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass.
Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.
Lead Product(s) : Olenasufligene Relduparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Undisclosed
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 : Undisclosed
Product Type : Cell & 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 : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass.
Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.
Lead Product(s) : Olenasufligene Relduparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Undisclosed
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 : Undisclosed
Product Type : Cell & 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 : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass.
Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.
Lead Product(s) : Olenasufligene Relduparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Undisclosed
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 : Undisclosed
Product Type : Cell & 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 : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass.
Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.
Lead Product(s) : Olenasufligene Relduparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Undisclosed
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 : Undisclosed
Product Type : Cell & 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 : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass.
Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.
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 : Undisclosed
Product Type : Cell & 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

Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass.
Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.
Lead Product(s) : Olenasufligene Relduparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Undisclosed
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 : Undisclosed
Product Type : Cell & 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 : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass.
Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.
Lead Product(s) : Olenasufligene Relduparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Undisclosed
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 : Undisclosed
Product Type : Cell & 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 : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass.
Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.
Lead Product(s) : Olenasufligene Relduparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Undisclosed
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 : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
June 05, 2020
Lead Product(s) : Olenasufligene Relduparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II/ Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass.
Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.
Lead Product(s) : Olenasufligene Relduparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Undisclosed
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 : Undisclosed
Product Type : Cell & 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 : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass.
Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.
Lead Product(s) : Olenasufligene Relduparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)
Details : LYS-SAF302 is a Cell and Gene Therapy drug candidate, which is currently being evaluated in phase II/ phase III clinical studies for the treatment of Mucopolysaccharidosis III.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
August 02, 2018
Lead Product(s) : Olenasufligene Relduparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II/ Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
