Latus Bio closes USD 97m Series A financing for AAV gene therapy pipeline

Philadelphia-based Latus Bio has [closed](https://www.businesswire.com/news/home/20260504989731/en/Latus-Bio-Announces-%2497-Million-Series-A-Financing-to-Expand-the-Reach-of-Gene-Therapy-to-Larger-Populations) a USD 97 million Series A financing round to advance its AAV gene therapy pipeline, with lead programs targeting Huntington's disease and a rare pediatric neurodegenerative disorder. The round's USD 43 million extension was led by 8VC, with existing investors DCVC Bio, BioAdvance, Benjamin Franklin Technology Partners, Modi Ventures, Gaingels, and Hatch BioFund participating alongside new investors Korea Development Bank and Helen's Pink Sky Foundation. The company said proceeds will fund operations through initial clinical data from its [two lead programs](https://www.businesswire.com/news/home/20260428641487/en/Latus-Bio-to-Present-New-Data-at-ASGCT-2026-Supporting-Investigational-Gene-Therapy-Approaches-for-Huntingtons-Disease-and-CLN2-Disease?utm_campaign=shareaholic\&utm_medium=copy_link\&utm_source=bookmark), LTS-201 for Huntington's disease and LTS-101 for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, as well as additional preclinical programs. The Series A was first opened in May 2024 at an initial close of USD 54 million, co-led by 8VC and DCVC Bio, bringing the total raised under the vehicle to USD 97 million. LTS-201 is an investigational AAV gene therapy encoding an engineered microRNA designed to reduce expression of MSH3, a DNA repair enzyme whose activity drives somatic repeat expansion in the mutant huntingtin gene. The company said an IND submission is expected in Q3 2026. LTS-101, targeting CLN2 disease, has [received IND clearance](https://www.businesswire.com/news/home/20251202268130/en/Latus-Bio-Announces-IND-Clearance-of-LTS-101-for-CLN2-Disease-and-Receipt-of-Fast-Track-Orphan-Drug-and-Rare-Pediatric-Disease-Designations) from the US FDA along with Orphan Drug, Rare Pediatric Disease, and Fast Track designations. An investigator-initiated first-in-human trial is expected to begin in Q3 2026, with initial safety, biomarker, and clinical data anticipated by Q4 2026. Beyond these two programs, Latus is advancing preclinical capsid variants with potential applications in kidney, eye, heart, and muscle diseases. The company's platform is built on proprietary AAV capsid variants developed through high-throughput, unbiased in vivo screening conducted in non-human primates. The scientific rationale centers on the premise that conventional AAV serotypes lack the tissue specificity required to achieve therapeutic transduction at clinically safe doses, particularly in the CNS. By screening tens of millions of capsid variants directly in non-human primates rather than rodents, the platform is designed to identify candidates whose performance is more likely to translate to human patients. Two named capsids have emerged from this process: AAV-DB-3, the basis for LTS-201, which demonstrated transduction of up to 45% of medium spiny neurons in the adult non-human primate striatum at doses estimated to be 10 to 100 times lower than those used with conventional capsids in the clinic; and AAV-Ep+, the basis for LTS-101, which targets ependymal cells and neurons following intracerebroventricular administration. The underlying science originates from the laboratory of [Beverly Davidson](https://app.allsci.com/researcher/ASC-PR-8965142738481-1.0-1765457341) at the [Children's Hospital of Philadelphia](https://app.allsci.com/organization/ASC-OH-0000000002994-1.0-1764853177) (CHOP), where the core capsid engineering research was conducted. CHOP has confirmed that related intellectual property has been licensed to Latus. Davidson serves as a paid consultant, holds equity in the company, and is a named inventor on the licensed IP. Both the AAV-DB-3 and AAV-Ep+ capsids were described in peer-reviewed publications in 2025, with the studies co-funded by the CHOP Research Institute and Latus. Davidson's laboratory is based within CHOP's Raymond G. Perelman Center for Cellular and Molecular Therapeutics. Latus is led by CEO P. Peter Ghoroghchian, MD, PhD, who the company describes as part of an experienced team of biotech and pharmaceutical leaders with expertise in gene therapy and CNS drug development. The company said it plans to pursue strategic partnerships to expand its platform for capsid discovery and to address non-core and non-CNS indications. Huntington's disease affects more than 100,000 patients across major gene therapy markets and currently has no approved disease-modifying therapies. The condition results from expansion of DNA repeats in the huntingtin gene, leading to progressive loss of medium spiny neurons in the striatum and projection neurons in the cortex. *** This article was generated with AI assistance and reviewed and edited by the AllSci editorial team Explore more at AllSci News: [https://allsci.com/news/](https://allsci.com/news/) --- Spot something wrong? 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