ATX-001 / Aclaris - LARVOL DELTA

Engineered AAV2 capsids have improved performance compared to natural serotypes via intra-CSF delivery in both mouse and NHP central nervous system (ASGCT 2026) - "Results When packaged, empty/full ratios of ATX001 and ATX002 are significantly improved compared to the natural AAV2 serotype, allowing for production of higher titers for CNS delivery...Furthermore, enhanced packaging of these capsids reduces the cost associated with manufacturing. These findings highlight the strength of Avista’s platform to generate highly manufacturable and potent next-generation AAV vectors for brain-wide gene therapy using lower doses via intra-CSF administration."

Preclinical • CNS Disorders • Gene Therapies

Molecular and Clinical Effects of Oral ATI-2138, an ITK/JAK3 inhibitor, in Moderate-to-Severe Atopic Dermatitis: Sub-study of a Phase 2a Open-Label, Single-Arm Trial (ISDS 2025) - P2 | "Treatment with ATI-2138 was associated with improvement in clinical measures and reductions in molecular markers of inflammation and fibrosis in skin and plasma. These findings suggest ATI-2138 as a new treatment for atopic dermatitis."

Clinical • P2a data • Atopic Dermatitis • Dermatitis • Dermatology • Fibrosis • Immunology • Inflammation • ADAMTS14 • CCL21 • CD4 • CD5 • COL1A1 • CXCL1 • CXCL12 • CXCL9 • IL13 • IL2 • IL33 • IL4R • IL6R • JAK3 • KRT7 • OASL • S100A9 • SPON1 • TGFB1 • TSLP

Molecular and Clinical Effects of Oral ATI-2138, an ITK/JAK3 inhibitor, in Moderate-to-Severe Atopic Dermatitis: Sub-study of a Phase 2a Open-Label, Single-Arm Trial (EADV 2025) - P2 | "Conclusion Treatment with ATI-2138 was associated with improvement in clinical measures and reductions in molecular markers of inflammation and fibrosis in skin and plasma. These findings suggest ATI-2138 as a potential new treatment for clinical and molecular reversal of atopic dermatitis."

Clinical • Late-breaking abstract • P2a data • Atopic Dermatitis • Dermatitis • Dermatology • Fibrosis • Immunology • ADAMTS14 • CCL21 • CD4 • CD5 • COL1A1 • CXCL1 • CXCL12 • CXCL9 • IL13 • IL2 • IL33 • IL4R • IL6R • JAK3 • KRT7 • OASL • S100A9 • SPON1 • TGFB1 • TSLP

Characterization of the dual ITK/JAK3 small molecule covalent inhibitor ATI-2138. (PubMed, J Pharmacol Exp Ther) - "ATI-2138 inhibits JAK3 and ITK in enzyme and functional cellular assays, demonstrates disease-modifying activity in rodent models of arthritis and colitis, and inhibits biomarkers linked to both ITK and JAK3 activity in healthy human participants. With this dual kinase activity against components of these inflammatory signaling pathway, ATI-2138 has the potential for enhanced therapeutic efficacy in the treatment of autoimmune and chronic inflammatory disease."

Clinical • Journal • Gastroenterology • Gastrointestinal Disorder • Immunology • Inflammation • Rheumatology • IL2 • ITK • JAK3