Our Pipeline
Programmes in Development
Transforma Bio is advancing a portfolio of gene and cell therapy programmes targeting rare, high-unmet-need diseases — alongside a dedicated nutraceutical and cosmeceutical research programme in sturgeon ECM.
Rare Monogenic Liver Disease
A recombinant AAV vector delivering a functional copy of the defective gene to hepatocytes, targeting a rare autosomal recessive metabolic disorder with no approved disease-modifying therapy.
Development Stage
Inherited Retinal Dystrophy
Subretinal delivery of a corrective gene construct targeting photoreceptor degeneration in a genetically defined patient population with progressive vision loss.
Development Stage
Haematologic Rare Disease
Ex vivo engineering of autologous haematopoietic stem cells using our proprietary cell engineering platform, targeting a rare blood disorder with significant morbidity.
Development Stage
Anti-Ageing (Oral Nutraceutical)
Oral formulation of purified sturgeon-derived ECM bioactives targeting systemic collagen renewal, skin hydration, and cellular anti-ageing markers.
Development Stage
Anti-Ageing (Topical Cosmeceutical)
Topical delivery system leveraging sturgeon ECM peptides and proteoglycans for dermal matrix restoration, targeting fine lines, elasticity loss, and photoageing.
Development Stage
Pipeline programmes are in early-stage research and development. Programme codes, indications, and timelines are subject to change. This information does not constitute an offer of securities or investment advice.
Partner With Us
Interested in our pipeline?
We welcome conversations with investors, strategic partners, and patient advocacy groups who share our commitment to transforming rare disease treatment.