[{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Sofinnova Investments","pharmaFlowCategory":"D","amount":"$55.0 million","upfrontCash":"Undisclosed","newsHeadline":"Atsena Therapeutics Raises $55 Million Series A Financing to Advance LCA1 Gene Therapy Clinical Program","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Atsena Therapeutics Receives Orphan Drug Designation from the FDA for Novel Gene Therapy to Treat Genetic Eye Disease LCA1","therapeuticArea":"Ophthalmology","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell 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Applicable","newsHeadline":"Atsena Therapeutics Announces Positive 12-month Safety and Efficacy Data from Ongoing Phase I\/II Clinical Trial of ATSN-101 in Patients with Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D (LCA1)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2023","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II"}]
Find Clinical Drug Pipeline Developments & Deals by Atsena Therapeutics
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).
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).
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
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.
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.
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).
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.
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.
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.
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.
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