Latus Bio, a Philadelphia-based developer of engineered AAV gene therapies for CNS disorders, has raised $97M in total Series A funding led by 8VC with participation from DCVC Bio. The startup's proprietary capsids enable 1/100 to 1/10,000 lower doses than competitors by precisely targeting brain and peripheral tissues. The capital, including an initial $54M tranche and $43M extension, will advance LTS-101 for CLN2 Disease toward clinical trials and LTS-201 for Huntington’s Disease.
Gene Therapy VC Dips Despite Approvals
Latus Bio's raise stands out as gene therapy venture funding fell to $773M across 20 rounds in 2024, down from $1.4B the prior year. The FDA approved 9 cell and gene therapies in 2024, signaling maturation. Competitors like Dyno Therapeutics raised $100M Series B in 2023 for AI-driven AAV design, while Affinia Therapeutics secured $110M previously for CNS programs. Latus differentiates through high-throughput NHP screens and AI/ML on 100 million delivery zip codes.
AAV Doses Trigger Toxicity Risks
Traditional AAV therapies require high doses for CNS penetration, leading to immune responses and liver toxicity that limit patient access. Monogenic CNS diseases like CLN2 Batten Disease affect rare populations with no cures. Current solutions fall short due to off-target effects and manufacturing hurdles. Engineered capsids address this by enhancing tropism.
Capsids Unlock Low-Dose CNS Delivery
Latus Bio's AAV.Ep+ capsid powers LTS-101, delivering TPP1 enzyme via cisterna magna for CLN2, with IND clearance, Fast Track, Orphan Drug, and Rare Pediatric designations from FDA. LTS-201 uses AAV.DB-3 to lower MSH3 and curb CAG expansions in Huntington’s basal ganglia. These outperform AAV5 in NHP models, per Nature Communications publication. AI/ML refines targeting from proprietary screens.
As P. Peter Ghoroghchian, Co-Founder & CEO, noted:
"This is an important step forward in our mission to bring scalable engineered gene therapies to larger numbers of patients."
Deep-Tech VCs Bet on Platform Scale
8VC, with $6B+ AUM and exits like Palantir IPO, led the extension alongside DCVC Bio, focused on computational biology. Samsung Life Science Fund adds manufacturing expertise. BioAdvance, Korea Development Bank, and foundations joined, signaling conviction in dose-sparing tech amid safety concerns.
Neurological Gene Therapy Market Expands
The gene therapy market for neurological diseases stands at $4B, growing at 13% CAGR. Overall gene therapy hit $5.54B in 2023, projected to reach $18.2B by 2030 at 18.9% CAGR. 4D Molecular Therapeutics went public in 2023 after productizing AAV capsids. Trends favor AI-enhanced vectors to cut toxicities.
Spark Pioneer Powers Breakthrough
Co-founder Beverly Davidson, SAB Chair, co-founded Spark Therapeutics, acquired by Roche for $4.3B after pioneering Luxturna, the first FDA-approved AAV gene therapy. CEO P. Peter Ghoroghchian founded Ceptur Therapeutics, raising $75M for RNA platforms. Recent hires include COO Ainslie Little from BlueRock Therapeutics and HD expert George Yohrling from Bristol Myers Squibb. This team bridges lab innovation to clinical execution.
Clinical Trials Follow IND Clearance
Latus plans LTS-101 clinical trials post-IND clearance, with LTS-201 advancing preclinical. AI/ML platform expansion targets broader diseases. $97M funds manufacturing scale-up and team growth to 50+ employees.