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Rare Treatment Accelerator

Our Rare Treatment Accelerator helps academic, patient, and industry groups advance their rare disease drug redevelopment projects. By combining our team, expertise and financial resources with your research and insights, we can get treatments to patients faster.

Supercharge your drug redevelopment project

Our Rare Treatment Accelerator (RTA) helps academic, patient, and industry groups advance their rare disease drug redevelopment projects. By combining our team, expertise and financial resources with your research and insights, we can get treatments to patients faster.

Who’s it for? 

The RTA is open to all academic, patient and early-stage biotech groups that have a drug redevelopment opportunity for a rare disease. Although different regions have their own definitions of a rare disease, we broadly define it as any condition that affects less than 1 person in 2,000 (5 in 10,000).

We’re particularly keen to hear about projects with strong evidence that the redeveloped drug has therapeutic effects in preclinical disease models and is suitable to move towards human clinical trials.

Why should I apply?

By applying our drug development and clinical expertise, and financial backing, we will help you progress your drug redevelopment project to a clinical trial much faster.

Apply here
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How we progress drug redevelopment opportunities

Speed

Speed

By applying our in-house expertise and technology to your drug redevelopment project we’re able to quickly plan and initiate clinical trials - within 6-12 months

Funding

Funding

We will manage the costs of clinical trials and regulatory approval for all selected drug redevelopment opportunities

Expertise

Expertise

The expertise of our drug development and commercial teams helps us evaluate, enhance and accelerate the development of your redevelopment projects

Application process

Interested? The first step is to complete a short eligibility questionnaire which will help us learn more about your work and disease area(s) of interest.

We review every questionnaire and contact everyone individually with feedback. 

If you have the necessary elements for your programme, we’ll invite you to complete a full application.

We are open to a range of collaboration opportunities, decided on a case-by-case basis. These include in-licensing of clinic-ready assets, option agreements with research funding and co-development for preclinical assets.

Please take a look at the FAQs below for more info on entry criteria and the selection process. For all other questions, please email our team at accelerate@healx.io.

Apply here
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Assessment process

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Partnerships

Icon FAQs

Redevelopment refers to starting with a drug, drug-like compound or nutraceutical already approved for human use in one or more approved indications, or has been shown in clinical trials to be safe for human use, and modifying it so that it can be used to deliver therapeutic benefit in one or more rare diseases for which the original drug or nutraceutical is not currently used.  

Examples of redevelopment strategies include changing the route of administration so it can hit a different tissue or organ, modifying the molecular structure so that more of the drug can reach the desired target, and combining two or more drugs in order to reduce the dosages and side effects of each while creating synergistic therapeutic effects.

The Rare Treatment Accelerator is our partnering programme for academic groups, patient groups and early-stage biotechs with promising drug redevelopment opportunities for rare or orphan diseases. Working together with selected applicants, we will leverage its financial resources, drug discovery and clinical expertise to accelerate novel therapies towards clinical trials.

The RTA programme gives participants access to our financial resources, drug discovery and clinical expertise needed to translate research into reality –  accelerating novel treatments and redeveloped drug candidates through clinical trials and towards patients.

The Rare Treatment Accelerator (RTA) is open to all academic, patient and biotech groups that have a promising drug redevelopment opportunity for a rare or orphan disease. Although different regions have their own definitions of a rare disease, we broadly define it as any condition that affects less than 1 person in 2,000 (5 in 10,000).

We are ideally looking for promising rare disease drug redevelopment opportunities with a strong preclinical package of data from validated models of the disease. “Promising” means that there is a good preclinical hypothesis and evidence that the redeveloped asset is effective in preclinical models that are representative of the rare disease(s). These diseases should also have validated clinical endpoints/outcome measures to enable translation to trials, as well as sufficient numbers of patients to enable clinical trials. We would also be looking for IP protection, or the opportunity for it, as well as the chance to create or enhance commercial viability through reformulation, combination or other modifications that also improve patient outcomes.

  • Open to all rare or orphan diseases (prevalence <5/10,000) with current unmet medical needs
    • Particularly interested in rare renal, inflammatory, neuromuscular, bone and ocular conditions
    • For rare oncology, we are not currently looking for opportunities relating to acute metastatic cancers with multiple genetic origins, such as acute leukemias, glioblastoma, neuroblastoma and similar conditions. However, we would be open to opportunities related to single gene-derived benign tumour types chronic cancers such as neurofibromatosis.
  • Ideally, the rare or orphan indication has:
    • Well-characterised natural history
    • Established clinical trial endpoints and/or biomarkers
    • Sufficient patients willing and able to participate in clinical trials
  • Basic through to clinical stage, with a particular interest in the late preclinical and clinical stages
  • Pre-clinically validated in models (in vitro and/or in vivo) that are representative of the disease of interest
  • Redevelopment* (using approved drugs) and repositioning (with shelved assets that were discontinued for non-safety reasons) of small molecules preferred; late-stage NCE candidates or nutraceuticals would also be considered
    • Gene therapy, biologics and cell therapy approaches are not of interest
  • 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

Reformulation refers to the development of different formulations for the same pharmaceutical drug to improve patient outcomes. For example, when sildenafil was repurposed for pulmonary arterial hypertension, sildenafil was reformulated from oral tablets (Viagra) to solution for injection, as well as powder for oral suspension (Revatio).

Whilst the Rare Treatment Accelerator is focused on accelerating promising drugs to clinical trials, we are still very interested in hearing from patient groups who want to work with us. Please fill in this form so the team can learn more about your work and disease area of interest, or reach out to Clara Tang, Alliance Strategy Manager, at accelerate@healx.io.

Interested groups for the Rare Treatment Accelerator can apply here.

There is no closing date for this Rare Treatment Accelerator. You can apply whenever you feel ready, starting with the eligibility questionnaire.

There is no cost to apply, and the collaboration to progress a clinic-ready asset for a particular disease will typically be at no cost to successful applicants. There may be times when we’ll agree with a partner to co-fund continuing pre-clinical work in preparation for bringing the asset to the clinical trial stage.

For this stage of the application, we ask groups to submit only non-confidential data. If  an application progresses to further review and due diligence, appropriate confidentiality agreements will be put in place if we need to evaluate confidential data as part of this process.

The Rare Treatment Accelerator panel is made up of our senior leadership team (including our Chief Medical Officer, Chief Scientific Officer and Chief Collaboration Officer) and members of our commercial, preclinical and clinical teams, who collectively hold over 100 years of drug discovery expertise in industry and not-for-profit organisations. 

If you have any questions, you can always reach out to our Alliance Strategy Manager, Dr Clara Tang, at accelerate@healx.io.

Our technology

Our AI leverages its unique combination of algorithms to predict and de-risk novel connections in biomedical data, and move the most promising ones towards the clinic.

About Healx

We’re a next-generation team of drug hunters, AI engineers and bioinformaticians, all working together to accelerate the discovery of new treatments for rare diseases.