Mapping the Contours of the T1D Therapeutic Landscape: Who's Ascending the Summit

Research evidence excerpt

Mapping the Contours of the T1D Therapeutic Landscape: Who's Ascending the Summit

BIOTECHNOLOGY Exhibit 2 - T1D Therapeutics Milestones/Catalysts

Source: Company Reports and BTIG ResearchINDUSTRY T1D Therapy Milestones/Catalysts

Near-term milestones include Lexicon Pharmaceuticals' sotagliflozin (Zynquista), with an NDA resubmission expected mid-2026,

while Zucara Therapeutics' ZT-01, has estimated primary completion of Ph1/2 ZONE study in 2H26. Further out, cell therapy

approaches are advancing with Sana Biotechnology's hypoimmune islet cell program SC451 targeting an IND application and Ph1REPORT trial start in late 2026, and NewCelX's human pluripotent stem cell program NCEL-101 targeting an IND submission in 1Q27. vTv

Therapeutics' cadisegliatin Ph3 CATT1 topline data are anticipated in 1H27. SAB Biotherapeutics' anti-thymocyte globulin program

SAB-142 rounds out the landscape with Ph2b SAFEGUARD topline data expected in 2H27.

Rising Therapeutic Modalities

Gene Editing/Therapy: The use of genetic material (or gene editing) to modify gene expression or cellular function for therapeutic

benefit. For T1D, gene editing aims to address (1) insulin deficiency by generating or reprogramming insulin-producing cells and (2)

autoimmune destruction of beta-cells by altering the immune pathways to promote tolerance. The most promising gene editing

concepts for T1D are those that either generate new insulin-producing cells, protect them from autoimmune attack, or both.

■ Adeno-associated Virus (AAV) Gene Delivery: The use of AAV vectors to deliver genes in vivo, typically aimed at increasing

insulin production, glucose sensing, or glucose regulation from non-pancreatic tissues. This approach bypasses the need