Arexera Therapeutics, Formerly Atalanta Therapeutics, Unveils New Corporate Identity And Advances Huntington's Disease Franchise

(MENAFN- GlobeNewsWire - Nasdaq) - Proprietary siRNA platforms, designed to enable robust and durable gene silencing throughout the brain, support a differentiated Huntington's disease franchise and broader neurological disease pipeline focused on genetically validated drivers of disease –

- ATL-101 preclinical data demonstrate robust and durable HTT suppression, including in the deep brain, with a potentially best-in-class profile; initiation of a Phase 1 study in Huntington's patients is anticipated by end of 2026 –

- Preclinical MSH3 programs address a complementary, genetically validated driver of Huntington's disease through potential first-in-class MSH3-targeting monotherapy and dual targeting of HTT and MSH3 –

BOSTON, Oct. 14, 2026 (GLOBE NEWSWIRE) -- Arexera Therapeutics, Inc. (“Arexera”), formerly Atalanta Therapeutics, a biotechnology company developing small interfering RNA (siRNA) medicines for severe neurological diseases, today unveiled a new name and corporate identity reflecting its new leadership team, refined pipeline focus and expanded platform capabilities as it prepares to enter the clinic with ATL-101, its lead program in Huntington's disease (HD).

“Over the past year, we have refined our strategic focus, assembled an experienced leadership team and expanded the capabilities of our siRNA platforms,” said Joanne Kotz, Ph.D., Chief Executive Officer of Arexera.“Huntington's disease is predominantly a disease of the deep brain, has well-established genetic drivers and a profound unmet need for treatments that change its course. We believe the robust, durable deep brain activity of our siRNA platforms provides strong differentiation and rationale for HD as an initial application. As Arexera, we are now preparing to enter clinical development with our lead program with the goal of developing disease-modifying therapies for HD patients.”

siRNA platforms offer significant differentiation and therapeutic advantages
RNA interference (RNAi) is a clinically validated therapeutic mechanism used by siRNA medicines to selectively reduce expression of target genes, but siRNA therapeutics have yet to realize their potential in many neurological diseases where achieving broad, durable activity across the central nervous system (CNS) remains a significant challenge. Arexera's proprietary divalent siRNA (di-siRNA) platform was designed to address these limitations by enabling robust and durable gene silencing throughout the brain, including deep brain structures.

In addition, Arexera's bispecific siRNA (bi-siRNA) platform is designed to target two distinct genes and is being evaluated preclinically. The Company is advancing a pipeline focused on genetically validated targets for which broad CNS distribution, robust target knockdown and durable activity create differentiated therapeutic opportunities.

Huntington's disease franchise strategy: advancing complementary approaches with best- and first-in-class potential
Arexera's HD franchise is built around two complementary, genetically validated drivers of disease: a disease-causing CAG repeat expansion in the huntingtin (HTT) gene, known as mutant huntingtin (mHTT); and MSH3, a key modifier of somatic CAG repeat expansion associated with disease onset and progression.

Arexera's lead program, ATL-101, is an investigational HTT-targeting di-siRNA therapeutic designed to reduce expression of HTT. In preclinical studies, ATL-101 demonstrated robust and durable HTT suppression throughout the brain, including in the striatum. The Company believes the combination of broad CNS distribution, robust striatal knockdown, prolonged durability and a favorable preclinical safety profile in multiple nonclinical models, including non-human primates (NHPs), positions ATL-101 as a differentiated, potentially best-in-class HTT-targeting therapy.

Arexera expects to initiate clinical development of ATL-101 by the end of 2026. The Phase 1 study will evaluate safety and changes in cerebrospinal fluid (CSF) biomarkers following single ascending doses of ATL-101 in Huntington's disease patients.

“Huntington's disease remains one of the most devastating neurodegenerative diseases, and there are currently no approved therapies that have been shown to slow or halt disease progression,” said Pedro Gonzalez-Alegre, M.D., Ph.D., Chief Medical Officer of Arexera.“ATL-101 has demonstrated a differentiated preclinical profile characterized by best-in-class HTT suppression in deep brain regions, prolonged durability and a favorable preclinical safety profile, attributes we believe may be important for achieving meaningful disease modification. We look forward to initiating our Phase 1 study in Huntington's disease patients and using the safety and biomarker findings to guide the program's next steps.”

In parallel, Arexera is advancing two therapeutic approaches targeting MSH3. Human genetic studies support MSH3 as a key modifier of disease progression, providing a rationale for investigating MSH3 lowering as an additional and complementary therapeutic approach. Arexera is currently developing a standalone MSH3-targeting di-siRNA and dual-targeting approaches for HTT and MSH3. One such dual-targeting approach would be the first application of its proprietary bi-siRNA platform. The Company is conducting parallel preclinical evaluations of both the mono- and dual-targeting strategies.

“By pursuing a potentially best-in-class approach to HTT lowering in ATL-101 alongside a di-siRNA targeting MSH3, we are building a Huntington's disease franchise designed to address complementary drivers of disease onset and progression,” added Bob D. Brown, Ph.D., President, Research & Development and Chief Scientific Officer.“We believe the dual HTT/MSH3-targeting approaches we are pursuing for HD can create a blueprint for combination approaches to treat other severe neurological diseases.”

Beyond HD, the Company has a preclinical-stage pipeline combining differentiated CNS distribution with genetically validated biology across additional neurological disease targets.

Arexera has assembled a management team with deep company-building experience and extensive expertise in RNAi and neurological drug discovery and development. To date, the Company has raised approximately $150 million to support advancement of its platform and pipeline.

About Huntington's Disease
Huntington's disease (HD) is a rare, fatal, inherited neurodegenerative disorder caused by an expanded CAG repeat in the huntingtin (HTT) gene. The disease progressively damages neurons in the brain and is characterized by motor, cognitive and psychiatric symptoms that worsen over time. HD typically presents between the ages of 30 and 50, with over 40,000 symptomatic patients in the United States and hundreds of thousands globally. There are no approved disease-modifying therapies for HD.

About Arexera Therapeutics
Arexera Therapeutics is a clinical-stage biotechnology company developing siRNA medicines for severe neurological diseases. The Company's proprietary divalent siRNA (di-siRNA) and bispecific siRNA (bi-siRNA) platforms are designed to enable robust and durable gene silencing throughout the brain, including deep brain regions that have historically been difficult to reach. Arexera is advancing a pipeline of therapies directed against genetically validated drivers of disease, with an initial focus on Huntington's disease, pursuing complementary approaches with ATL-101, targeting HTT, and MSH3-directed strategies, as well as future opportunities across other severe neurological diseases. The Company is also advancing partnered programs as part of a strategic collaboration with Genentech. Arexera is headquartered in Boston, Mass. For more information, visit .

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