Gene Medicines — Advancing the Next Generation of Non-Viral Curative Therapies
The core long-term strategic purpose of the HPAE platform is enabling safe, accessible genetic medicines for patients living with rare untreatable inherited diseases. Every polymer advancement we develop is engineered to accelerate our clinical pipeline programmes.
Our core therapeutic vision
Our pipeline strategy prioritises rare monogenic disorders with clearly defined molecular root causes, where non-viral HPAE delivery can deliver repeated, low-toxicity therapeutic genetic cargo without the immunogenic risks of viral vector systems. Our work aims to expand global patient access to durable genetic correction treatments.
Scientific discovery
Targeted gene delivery
Tissue repair
Improved patient outcomes
Six platform therapeutic modalities
Gene Replacement Therapy
Deliver full-length functional transgenes to restore missing protein expression (BRB202 core programme).
CRISPR Gene Editing
Targeted permanent correction of pathogenic gene mutations (BRB101 core programme).
mRNA Transient Therapeutics
Temporary protein expression for acute tissue repair applications.
Ex Vivo & In Vivo Cell and Gene Therapy Support
Non-viral delivery chemistry adaptable to both ex vivo and in vivo therapeutic workflows.
Tissue Regenerative Genetic Delivery
Localised genetic cargo delivery supporting tissue repair and regeneration.
Future Emerging Modalities
Continuous platform expansion into new therapeutic categories as R&D matures.
HPAE
Core platform
Skin — Dystrophic Epidermolysis Bullosa
Oncology genetic programmes
Regenerative medicine
Immunotherapy delivery
Dystrophic Epidermolysis Bullosa — flagship proof-of-concept
DEB is a severe heritable skin fragility disorder caused by COL7A1 gene mutations, resulting in absent or defective type VII collagen anchoring fibrils, chronic blistering, scarring and elevated skin cancer risk. Two complementary HPAE therapeutic strategies are in pre-clinical development to address this unmet clinical need.
CRISPR Exon Deletion Gene Editing Therapy
- EMA Orphan Drug Designation
- Permanent targeted correction of the mutant COL7A1 gene in skin keratinocytes and fibroblasts
- Repeat-administrable non-viral delivery platform
Scientific diagram: HPAE polymer + Cas9/sgRNA RNP polyplex formation and in-vivo skin tissue editing pathway
Full-Length COL7A1 Gene Replacement Therapy
- EMA Orphan Drug Designation
- Delivers an intact functional COL7A1 transgene to restore type VII collagen fibril formation
- Optimised for chronic wound local delivery
Scientific diagram: HPAE + full-length COL7A1 plasmid polyplex delivery to dermal tissue
Modular architecture, expanding therapeutic reach
While DEB is our flagship translational pipeline asset, the modular HPAE architecture supports broad expansion across oncology, regenerative medicine and immune genetic therapies as our R&D programmes mature.
Co-develop HPAE-enabled therapeutic programmes
Branca Bunús actively seeks pharma, biotech and academic clinical partners to co-develop HPAE-enabled gene therapy programmes across all therapeutic categories. We offer flexible platform licensing, joint development and pre-clinical collaboration frameworks for aligned strategic partners.