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ATSN-201 is an investigational gene therapy that leverages one of the company’s novel spreading capsids, AAV.SPR, it is being developed for the treatment of X-linked retinoschisis (XLRS).
Lead Product(s): ATSN-201
Therapeutic Area: Genetic Disease Product Name: ATSN-201
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 March 13, 2024
Details:
SAR439483 (ATSN-101) is a novel GUCY2D modulator which is currently being evaluated in phase 1/2 clinical development for the treatment of GUCY2D-associated leber congenital amaurosis (LCA1).
Lead Product(s): ATSN-101,Prednisone,Triamcinolone
Therapeutic Area: Rare Diseases and Disorders Product Name: SAR439483
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 January 16, 2024
Details:
ATSN-101 is a LCA1 gene Transference Cell and Gene therapy drug candidate, which is currently being evaluated for the treatment of patients with GUCY2D-mutated Leber Congenital Amaurosis.
Lead Product(s): ATSN-101,Prednisone,Triamcinalone Acetonide
Therapeutic Area: Rare Diseases and Disorders Product Name: ATSN-101
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 04, 2023
Details:
ATSN-101, gene therapy for patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1). RMAT designation was granted based on positive 6-month efficacy data from the company’s ongoing Phase I/II clinical trial of ATSN-101.
Lead Product(s): ATSN-101
Therapeutic Area: Rare Diseases and Disorders Product Name: ATSN-101
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 November 14, 2023
Details:
ATSN-201 leverages AAV.SPR, the company’s novel spreading capsid, to achieve therapeutic levels of gene expression in photoreceptors of the central retina while avoiding the surgical risks of foveal detachment.
Lead Product(s): ATSN-201
Therapeutic Area: Genetic Disease Product Name: ATSN-201
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 28, 2023
Details:
ATSN-201 is an investigational gene therapy that leverages one of the company’s novel spreading capsids, AAV.SPR, for the treatment of X-linked retinoschisis (XLRS).
Lead Product(s): ATSN-201
Therapeutic Area: Genetic Disease Product Name: ATSN-201
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 May 01, 2023
Details:
SAR439483 (ATSN-101) is an investigational gene therapy which is being evaluated for patients with GUCY2D-associated Leber congenital amaurosis (LCA1), a monogenic eye disease that disrupts the function of the retina.
Lead Product(s): ATSN-101
Therapeutic Area: Rare Diseases and Disorders Product Name: ATSN-101
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 April 25, 2023
Details:
SAR439483 (ATSN-101) is being evaluated for patients with GUCY2D-associated Leber congenital amaurosis (LCA1), a monogenic eye disease that disrupts the function of the retina.
Lead Product(s): ATSN-101
Therapeutic Area: Rare Diseases and Disorders Product Name: SAR439483
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 08, 2023
Details:
Data demonstrated that subretinal delivery ATSN-101 (SAR439483) was well tolerated and patients treated with highest dose clinically meaningful improvements in vision, as measured by full-field stimulus testing and multi-luminance mobility testing.
Lead Product(s): ATSN-101
Therapeutic Area: Rare Diseases and Disorders Product Name: SAR439483
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 October 03, 2022
Details:
Subretinally injected AAV.SPR enables ATSN-201 to overcome the challenges associated with intravitreally delivered AAVs in the treatment of XLRS and facilitates safe delivery of RS1 to photoreceptors in the central retina/fovea.
Lead Product(s): ATSN-201
Therapeutic Area: Genetic Disease Product Name: ATSN-201
Highest Development Status: IND EnablingProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 02, 2022
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