Our Science
A Platform for Transformative Therapies
We combine gene delivery engineering, cell therapy, and translational science to build a pipeline of treatments for patients with rare and serious diseases.
Platform Overview
How We Work
Transforma Bio's platform is built on three interlocking capabilities — precision gene delivery, engineered cell therapies, and a rigorous translational pipeline. Together, they allow us to move from target identification to clinical candidate faster, and with greater confidence in safety and efficacy.
Gene Delivery
Precision at the Molecular Level
Our proprietary delivery vectors are engineered for precision — targeting the right cells, in the right tissue, with minimal off-target effects. We develop both viral and non-viral delivery systems, optimised for each therapeutic indication.
Capabilities
- Viral vector engineering (AAV, lentiviral)
- Lipid nanoparticle (LNP) delivery systems
- Tissue-specific targeting strategies
- Minimised immunogenicity profiles
Cell Engineering
Living Therapies That Last
We engineer patient-derived cells to carry corrective genetic payloads, creating living therapies that can persist and function long-term. Our cell engineering platform spans ex vivo modification, quality control, and scalable manufacturing.
Capabilities
- Ex vivo cell modification protocols
- CAR-T and TCR-T engineering
- Haematopoietic stem cell (HSC) editing
- GMP-compatible manufacturing processes
Translational Science
From Bench to Bedside
Our translational team bridges discovery and clinical development — building the preclinical evidence packages, biomarker strategies, and regulatory frameworks needed to accelerate the path to patients.
Capabilities
- Disease model development and validation
- Biomarker identification and assay development
- Regulatory strategy and IND preparation
- Clinical trial design and site partnerships
Sturgeon ECM
Nature's Blueprint for Anti-Ageing
Transforma Bio harnesses the extraordinary regenerative properties of sturgeon-derived extracellular matrix (ECM) — a bioactive scaffold rich in collagen, proteoglycans, and growth factors. In the body, ECM proteins such as Vitronectin, Fibronectin, and Laminin regulate cell adhesion, self-renewal, and tissue homeostasis through integrin-mediated signalling. Our R&D programme isolates and characterises these bioactive components from sturgeon, developing both ingestible and topical formulations that work at the cellular level to restore skin architecture and slow the hallmarks of biological ageing.
Sturgeon ECM — Bioactive Proteins
The Science Behind the Matrix
Extracellular matrix proteins regulate cell adhesion, self-renewal, and tissue homeostasis through integrin-mediated signalling. Transforma Bio's sturgeon ECM programme isolates and characterises these bioactive components for therapeutic and cosmeceutical applications.
Vitronectin
A small-molecular-weight glycoprotein present in serum and ECM that promotes cell adhesion, spreading, and migration via αvβ5 and αvβ1 integrin interactions. Supports stem cell pluripotency and normal karyotype maintenance.
Fibronectin
A large extracellular glycoprotein with multiple binding domains that regulates cell migration, adhesion, proliferation, and wound healing. Promotes stem cell self-renewal and can direct differentiation towards cardiac and pancreatic lineages.
Laminin
A cross-shaped trimeric protein (α, β, γ chains) and the primary component of the basement membrane. Laminin-511 and Laminin-521 are expressed from the 2–4 cell embryonic stage and regulate stem cell proliferation, self-renewal, and multi-lineage differentiation via FAK, AKT, and YAP signalling.
Collagen
The most abundant ECM protein, providing tensile strength and structural integrity to tissues. Sturgeon-derived collagen offers a non-mammalian, low-immunogenicity source with high biocompatibility for cosmeceutical and nutraceutical applications.
Proteoglycans
Heparan sulphate and chondroitin sulphate proteoglycans regulate growth factor gradients, water retention, and cell signalling within the ECM niche — key contributors to skin hydration and elasticity.
ECM Niche Context
Stem cells depend on a chemically defined, reproducible ECM microenvironment for stable expansion and controlled differentiation. Transforma Bio's sturgeon ECM research aims to provide xeno-free, batch-consistent bioactive matrices for both therapeutic and cosmeceutical use.
Research Capabilities
- Sturgeon ECM extraction and purification
- Oral bioavailability and absorption studies
- Topical delivery and skin penetration research
- Anti-ageing efficacy and safety validation
- Nutraceutical and cosmeceutical formulation
Scientific References — Sturgeon ECM
- 1Hynes RO. The extracellular matrix: not just pretty fibrils. Science. 2009;326(5957):1216–1219.
- 2Abdal Dayem A, et al. The Impact of Adhesion Molecules on the In Vitro Culture and Differentiation of Stem Cells. Biotechnol J. 2018;13(2).
- 3Braam SR, et al. Recombinant vitronectin is a functionally defined substrate that supports human embryonic stem cell self-renewal via αvβ5 integrin. Stem Cells. 2008;26(9):2257–2265.
- 4Zhang J, et al. Cardiac differentiation of human pluripotent stem cells using defined extracellular matrix proteins reveals essential role of fibronectin. Elife. 2022;11.
- 5Rodin S, et al. Long-term self-renewal of human pluripotent stem cells on human recombinant laminin-511. Nat Biotechnol. 2010;28(6):611–615.
- 6Aumailley M. The laminin family. Cell Adh Migr. 2013;7(1):48–55.
Our Approach
Why Rare Diseases?
Rare diseases represent one of medicine's greatest unmet needs. With over 7,000 known rare diseases and 95% lacking an approved therapy, the opportunity to make a meaningful difference is immense. Gene and cell therapies are uniquely suited to address the root causes of many rare conditions — and Transforma Bio is building the platform to deliver them.
7,000+
Known rare diseases worldwide
95%
With no approved therapy
300M+
People affected globally
See Our Pipeline
Our platform is generating a pipeline of gene and cell therapy candidates across multiple rare disease indications.
View Pipeline