Drugs in Dev. Genetic Disease
Phase II/ Phase III
France 
Lead Product(s) : Venglustat
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Sanofi Halts Venglustat Clinical Programme in ADPKD
Details : The STAGED-PKD study was stopped for futility following an independent analysis of the annualized rate of change in total kidney volume (TKV) in patients receiving venglustat compared to placebo.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
June 01, 2021
Lead Product(s) : Venglustat
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II/ Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Venglustat
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Venglustat is a Other Small Molecule drug candidate, which is currently being evaluated in phase II/ phase III clinical studies for the treatment of Polycystic Kidney, Autosomal Dominant.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
May 14, 2018
Lead Product(s) : Venglustat
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II/ Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
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
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
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
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
Lead Product(s) : QR-421a
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Sepul Bio
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : QR-421a is a Oligonucleotide drug candidate, which is currently being evaluated in phase II/ phase III clinical studies for the treatment of Retinitis Pigmentosa.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
January 04, 2022
Lead Product(s) : QR-421a
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II/ Phase III
Sponsor : Sepul Bio
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Ultevursen
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Sepul Bio
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Ultevursen is a Oligonucleotide drug candidate, which is currently being evaluated in phase II/ phase III clinical studies for the treatment of Retinitis Pigmentosa.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
December 15, 2021
Lead Product(s) : Ultevursen
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II/ Phase III
Sponsor : Sepul Bio
Deal Size : Inapplicable
Deal Type : Inapplicable
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
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
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