Articles from Atsena Therapeutics

First XLRS study to demonstrate efficacy and positive safety data in a Phase I/II trial

DURHAM, N.C., April 29, 2025 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on using the life-changing power of genetic medicine to reverse or prevent blindness, today announced it will present at the following scientific conferences in May:

Marks fourth FDA designation for ATSN-201, which has also received Fast Track, Rare Pediatric Disease and Orphan Drug Designations

Financing led by new investor Bain Capital with participation from new investor Wellington Management and all existing investors

Marks third FDA designation for ATSN-201, which has also received Rare Pediatric Disease Designation and Orphan Drug Designation

DURHAM, N.C., Feb. 06, 2025 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced that safety and efficacy data on ATSN-201 for the treatment of X-linked retinoschisis (XLRS) will be presented during a panel discussion at The Macula Society’s 48th Annual Meeting taking place February 12-15, 2025, in Charlotte Harbor, Florida. ATSN-201, a best-in-class gene therapy product candidate, 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.

Part B of the LIGHTHOUSE study will enroll adults and children

DURHAM, N.C., Dec. 16, 2024 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced dosing has been completed in Part A of the LIGHTHOUSE study, a Phase I/II clinical trial evaluating subretinal injection of ATSN-201 for the treatment of X-linked retinoschisis (XLRS). ATSN-201, a best-in-class gene therapy product candidate, 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.

KYOTO, Japan and DURHAM, N.C., Nov. 13, 2024 (GLOBE NEWSWIRE) -- Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; Headquarters: Kyoto; President, Toru Nakai) and Atsena Therapeutics, Inc. (Atsena; Headquarters: Durham, North Carolina, USA, Chief Executive Officer (CEO): Patrick Ritschel) have entered into an exclusive license agreement for the commercialization of ATSN-101 in the territory of the U.S. and for the development and commercialization of ATSN-101 in the territory of Japan for advancing Atsena’s first-in-class, investigational gene therapy ATSN-101 for Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1).

DURHAM, N.C., Oct. 10, 2024 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced that safety and efficacy data on ATSN-201 for the treatment of X-linked retinoschisis (XLRS) will be presented during a panel discussion at the American Academy of Ophthalmology (AAO) 2024 Annual Meeting taking place October 18-21, 2024, in Chicago. ATSN-201, a best-in-class gene therapy product candidate, 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.

DURHAM, N.C., Sept. 24, 2024 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced that it will present at Chardan’s 8th Annual Genetic Medicines Conference taking place September 30 - October 1, 2024, in New York City. Chief Executive Officer Patrick Ritschel will deliver a company presentation on Tuesday, October 1, 2024, at 11:30 a.m. ET. Atsena Chief Medical Officer, Kenji Fujita, MD, and Mr. Ritschel will also participate in one-on-one meetings with potential investors during the conference.

Marks second FDA designation for ATSN-201, which previously received Rare Pediatric Disease designation

First time patients with LCA1 have been treated with gene therapy

Priority Review Voucher would be issued upon approval of ATSN-201

Biotech and pharma veteran brings expertise in therapeutic development and commercialization

DURHAM, N.C., May 01, 2024 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced positive preliminary data from the first cohort of the ongoing LIGHTHOUSE study, a Phase I/II clinical trial evaluating subretinal injection of ATSN-201 for the treatment of X-linked retinoschisis (XLRS). ATSN-201 utilizes 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.

DURHAM, N.C., March 13, 2024 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced dosing has been initiated in the second cohort of the LIGHTHOUSE study, a Phase I/II clinical trial evaluating subretinal injection of ATSN-201 for the treatment of X-linked retinoschisis (XLRS). ATSN-201, a best-in-class gene therapy product candidate, 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.

Positive 12-month safety and efficacy data from ongoing Phase I/II clinical trial of ATSN-101 to be presented at 47th Annual Macula Society Meeting on February 7, 2024

DURHAM, N.C., Dec. 04, 2023 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced positive 12-month safety and efficacy data from the ongoing Phase I/II trial of ATSN-101, the company’s investigational gene therapy for the treatment of GUCY2D-associated Leber congenital amaurosis (LCA1). At 12 months post-treatment, ATSN-101 has conferred clinically meaningful improvements in vision at the highest dose with no serious treatment-emergent adverse events.

RMAT designation recognizes the potential of ATSN-101 to address unmet medical needs for patients with LCA1

The LIGHTHOUSE study evaluating the safety and tolerability of investigational gene therapy ATSN-201 is enrolling male patients ages 6-64 with RS1-associated XLRS

ATSN-201 leverages AAV capsid that spreads laterally beyond subretinal injection site to facilitate safe delivery of RS1 to photoreceptors in the central retina/fovea

DURHAM, N.C., April 25, 2023 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced positive 6-month safety and efficacy data from the ongoing Phase I/II clinical trial of ATSN-101, the company’s investigational gene therapy for the treatment of GUCY2D-associated Leber congenital amaurosis (LCA1). The data were presented by Christine Nichols Kay, MD, Clinical Ophthalmology Advisor for Atsena, at the Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting.

DURHAM, N.C., April 11, 2023 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced that 6-month safety and efficacy data from the ongoing Phase I/II clinical trial of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1) will be presented at the Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting, which is being held April 23-27 in New Orleans, LA.

ATSN-101 has demonstrated clinically meaningful improvements in vision with no drug-related serious adverse events

DURHAM, N.C., Dec. 07, 2022 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced that Patrick Ritschel, Chief Executive Officer, will present a company overview at the Emerging Life Science Company Showcase hosted by NewYorkBIO and NYSE. The presentation will take place at The New York Stock Exchange on Wednesday, December 14, 2022, at 4:15 p.m. EST.

Biotech leader brings extensive experience in finance, business development and corporate strategy

ATSN-101 demonstrated clinically meaningful improvements in vision with no drug-related serious adverse events

World-class facility supports the company’s growth, and discovery and development of gene therapies for inherited retinal diseases