Therapy Assets Call
Advance your rare disease drug redevelopment projects
Let's accelerate treatments to the rare disease community
Blend our team, expertise and financial resources with your research and insights, so we can get treatments to patients faster.
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.
What are we looking for?
We are 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.
Step 1: Complete eligibility questionnaire
Step 2: Sign CDA to share further information
Step 3: Submit full application
Step 4: Negotiation and closing of deal
Step 5: Project starts
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.
We give selected 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.
We are 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 looking for therapeutic opportunities for rare or orphan diseases with a strong preclinical package of data. Redevelopment (using approved drugs) and repositioning (with shelved assets that were discontinued for non-safety reasons) of small molecules (monotherapies or combinations) are preferred, but late-stage NCE candidates or nutraceuticals would also be considered. Gene therapy and cell therapy approaches are not of interest.Ideally, patent protection will be 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.
We are open to all rare or orphan diseases (prevalence <5/10,000) with current unmet medical needs, with a particular interest 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, your 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
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 we are 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 email@example.com.
Interested groups can apply here.
There is no closing date. 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 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 firstname.lastname@example.org.