Seeking Opportunities for the Treatment of Rare or Orphan Diseases

Healx, an AI-powered, patient-inspired, biotech company, is seeking rare disease therapeutic opportunities from academic, patient groups or (early-stage) biotechs. Healx, along with their partners, aim to quickly turn research into treatments that benefit the rare disease community, and Healx will manage the costs of clinical trials and regulatory submissions for all selected opportunities (subject to final deal terms).

Approaches of Interest

• Open to all rare or orphan diseases (prevalence <5/10,000) with current unmet medical needs, with a particular interest in rare neurodevelopmental, neuromuscular, renal, liver, ophthalmology, and bone diseases, including diseases already in Healx’s pipeline
• Ideally, the rare or orphan indication has a well-characterised natural history, established clinical trial endpoints and/or biomarkers and, if applicable, sufficient patients willing and able to participate in clinical trials

Stage of Development

• Healx is open to opportunities at the late preclinical and clinical stages
• Preclinical validation in models (in vitro and/or in vivo) that are representative of the disease of interest is required

Ideal Candidate Profile

• Drug redevelopment (modifying an approved drug) repositioning (with shelved assets that were discontinued for non‑safety reasons), late-stage new chemical entity (NCE) candidates, simple biologics (e.g., peptides) or nutraceuticals
• Patent protection available or already prosecuted (composition of matter, method of use and/or formulation patents)
• For opportunities redeveloping approved drugs or nutraceuticals: there needs to be a chance to create or enhance both patient impact and commercial viability through reformulation, combination or other modifications that also improve patient outcomes and manage “off‑label” use risk

Out of Scope

• Antisense oligonucleotides, cell or gene therapies are not of interest
• For rare oncology, Healx are not currently looking for opportunities relating to acute metastatic cancers with multiple genetic origins e.g., acute leukaemias, glioblastoma, and neuroblastoma. Infectious diseases are also deprioritised 

Opportunity for Collaboration and Submission Information

• Healx is open to a range of collaboration opportunities, with the most appropriate outcome being decided on a case-by-case basis. Example outcomes include in‑licensing clinic‑ready assets for clinical development, option agreements with research funding and co‑development for preclinical assets
• To submit your research, please fill in the submission form. In submitting to this campaign, you confirm that your submission contains only non‑confidential information