ALY-101 / Alys Pharma - LARVOL DELTA

Disease severity and rate of progression impact ex-vivo gene therapy for Childhood Cerebral Adrenoleukodystrophy and Metachromatic Leukodystrophy: a review of the literature (ASGCT 2026) - P1/2, P2, P2/3, P3 | "For both disorders the FDA has approved ex-vivo lentiviral gene therapy: elivaldogene autotemcel (eli-cel; lentiviral vector carrying ABCD1 cDNA under a MNDU3 promoter-enhancer) and atidarsagene autotemcel (arsa-cel; lentiviral vector carrying ARSA cDNA under PGK promoter)...Results Adrenoleukodystrophy In the eli-cel trials 67 CCALD patients were treated (32 in ALD-102 with cyclophosphamide/busulfan conditioning and 35 in ALD-104 with fludarabine/busulfan conditioning)...As hematological malignancies were seen with eli-cel but not arsa-cel, treatment with eli-cel should be limited to CCALD patients who do not have a suitably matched donor available for allogeneic HSCT. AEs related to conditioning and disease were important contributors to overall safety in both trials."

Gene therapy • Preclinical • Review • Acute Myelogenous Leukemia • Alzheimer's Disease • Bone Marrow Transplantation • CNS Disorders • Cognitive Disorders • Developmental Disorders • Gene Therapies • Genetic Disorders • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Metabolic Disorders • Myelodysplastic Syndrome

Alys Pharmaceuticals Announces Dosing of First Patient in Phase IIa Trial of ALY-101 for Alopecia Areata (PRNewswire) - "Alys Pharmaceuticals...today announces that the first patient has been dosed in its Phase IIa trial of ALY-101 in patients suffering from Alopecia Areata (AA), a chronic autoimmune disease with significant unmet medical need that often leads to significant psychological distress and a reduced quality of life....The Phase IIa trial will investigate if ALY-101 is safe and well tolerated following injections in the scalp in subjects with alopecia areata randomly vs. placebo every 4 weeks for a treatment period of 8 weeks. The study is expected to be completed in 2026."

P2a data • Trial status • Alopecia

Alys Pharmaceuticals receives IND clearance for Phase 2a trial of first genetic medicine program in dermatology (PRNewswire) - "Alys Pharmaceuticals, Inc...announces that the U.S. Food and Drug Administration (FDA) and Health Canada have cleared the Investigational New Drug (IND) / Clinical Trial Application (CTA) for the study of ALY-101 in patients suffering from Alopecia Areata (AA). The Phase 2a trial will evaluate the safety and efficacy of ALY-101, an intradermal injectable JAK1 siRNA-lipid conjugate, at five sites across the United States and Canada...The Company remains on track to deliver multiple clinical Proof-of-Concept (POC) readouts by 2027."

IND • Alopecia