Rare Treatment Accelerator
Working together we can find treatments for rare diseases – fast. The Rare Treatment Accelerator offers patient groups the chance to receive up to the value of $1 million, in AI and drug discovery resources, to develop new therapies
Programme now closed for entriesRegister interest
A rare opportunity
The Rare Treatment Accelerator is a partnering programme that gives patient groups and Healx the opportunity to work together to quickly discover and develop repurposed treatments for rare diseases using artificial intelligence (AI).
We have committed a total of $20 million over the next two years towards finding new treatments – investing up to the value of $1 million, in AI and drug discovery resources per project. Working together, with selected applicants, we’ll combine our AI technology, data, disease and drug discovery expertise to develop novel therapies and take them towards clinical trials in a typical timeframe of 24 months.
We believe in long-term partnerships where everyone brings the determination, expertise and the resources needed to succeed in driving patient access to therapies. Sound good to you? Find out more and register your interest below.Register interest
Dr Bruce Bloom
Patient groups, patients and carers have insights, resources and relationships that can multiply the speed and value of our AI therapy discovery. We need each other to make quick progress for rare disease patients.
Dr Michael Tranfaglia
The Healx partnership has been nothing short of amazing. In less than two years together, we were able to deliver decades-worth of drug discovery, and now we’re taking those discoveries to the clinic.
Dr Rick Thompson
Healx combine a deep knowledge of drug repurposing with a passionate desire to deliver real change to rare disease patients.
Dr Cesare Spadoni
Healx changed my perception of what's possible in drug development.
Register your interest
The Rare Treatment Accelerator is now closed for entries. Thanks to everyone who applied! We have been impressed with the number and quality of applications received and are now in the process of assessing entries.
To receive the latest updates on the Rare Treatment Accelerator, please click below to register your interest.
Have any questions? Take a look at our handy FAQs section or email us at firstname.lastname@example.org if you can’t find the information you’re after.Register interest
The Rare Treatment Accelerator is open to patient groups worldwide that focus on rare diseases. Umbrella organisations that support more than one rare disease can help by encouraging their supported patient groups to participate. All patient groups must represent patients and be registered as a charity or foundation.
Although the Rare Treatment Accelerator is not open to industry partners, we pursue licensing and co-development partnerships with industry to translate new treatments towards the clinic.Learn more
We’ve already used our technology platform to help other patient groups successfully identify repurposed candidate drugs and get them ready for clinical trials within 24 months of starting a project. We want to use our AI platform Healnet to accelerate and de-risk the drug discovery process with additional patient groups, so we can get new treatments faster and cheaper to those who need it most.
The Rare Treatment Accelerator (RTA) programme is open to rare disease-specific patient groups and umbrella organisations worldwide.
Unfortunately, the RTA programme is aimed at registered patient groups and/or umbrella organisations. The best way for patients and carers to participate is through one of these organisations. If you’re a rare disease patient or carer and need support in organising or contacting a disease-specific charity, please reach out to a rare disease umbrella group such as Findacure, Eurordis, Global Genes, Canadian Organization of Rare Diseases, NORD or Rare Disease Foundation Canada.
We’re eager to partner with as many collaborators as possible around individual rare diseases. We would ask collaboration groups to designate a lead charity/project leader to submit the application and, if the disease is selected, to contract with us.
Umbrella charities can help by sharing the RTA programme with the specific rare disease patient charity groups they represent. If an umbrella charity wants to apply on its own, it will have to submit a separate application for each disease.
We’re open to working with patient groups worldwide.
There is no cost to apply, and the partnership will be at no cost to selected patient groups.
If there are many groups involved in supporting one disease, it is fine for you to provide that information, even if it doesn’t fit nicely into the boxes provided for the answers. In the case cited above, it would be fine to write, “There are at least 50 patient groups we know of working in this disease. We work with ______ and _____, and do not have a working relationship with the others.”.
For the application, we’re asking you to submit only non-confidential data. If and when we agree on a collaboration, there may be a mutual exchange of confidential information, at which point we (both parties) will sign the necessary confidentiality agreements so that we can quickly start working towards finding treatments for the patients.
We know it may be a challenge for some patient groups to complete the application by the 10th January 2020 deadline, depending on the size of the staff and the availability of experts whose support you need. In case you have missed the deadline, please do email us at email@example.com We plan to partner with at least 20 patient groups over the next two years, and we plan to have regular requests for applications over that time period.
If you’re having trouble submitting online, please contact us at firstname.lastname@example.org and someone from our team will be in touch with you to help.
The Rare Treatment Accelerator panel is made up of our senior leadership team (to include our CMO, CSO and COO), who collectively hold over 90 years of drug discovery expertise in industry and not-for profit organisations https://healx.io/about/#leadership
The application is designed to give us a comprehensive look at your organisation, rare disease of interest, network and other resources. For a collaboration to have the greatest potential, we’ll need sufficient, relevant data to apply in our AI platform, and we’ll have to be able to use multiple drug-matching methods so we can cross-validate and get more robust drug predictions.
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) and whose symptoms have unmet medical needs.
We choose diseases which have characteristics that make it more likely that patient outcomes can be improved by the therapeutic application of repurposed monotherapies and drug combinations. Each disease has its own characteristics, and it is the combination of these characteristics that helps us select. Some of the characteristics we look for are how many patients have the disease, when does it first manifest itself, how long does it last, what is the course of the disease, do we know the genetic or other causes, do we know the pathways and targets involved, do we have any biomarkers or other ways to judge disease progression or disease resolution, what are the signs and symptoms and how might they be managed, what therapies are currently given and how do they work, what other therapies have been tried and failed, what therapies are in development, how are patients able to participate in research, are there validated preclinical models, is there data available that would strengthen our predictions, are there validated outcome measurements, are there validated quality of life and other scales used to evaluate patients, are there similar diseases that have therapies, are there networks of scientists and clinicians that are invested in the disease and work together, and many more characteristics.
The most important thing a patient group can do is share with Healx what they know about their disease(s) of interest, the current therapies and groups working on new therapies, who they are connected to, and what models, tools and resources are available. This information gives our team at Healx the ability to determine how we might be able to accelerate repurposed therapy delivery to patients by working together. What isn’t in the application is the patient groups willingness and ability to help. We have had amazing patient group partners from our work in fragile X and Pitt Hopkins, and a lot of the success was that both the Healx team and the patient group team were open, hard-working, trusting, curious, forgiving, and persistent. Healx knows we need to give 100% to each collaboration, and we will look for patient group partners that feel the same.
This depends on the stage of the project we’re in, but to put things simply, this partnership comes at no cost to the patient group, except for the time it takes you to help us work with your resources. We’re looking for expertise, information, advice, and perhaps most importantly, we’re looking for engaged and collaborative partners.
We have been working with several patient groups to design an agreement. Once we have completed the drafting we can share it with any patient group that has submitted an application that has been evaluated by our team as complete and likely to create a good collaboration.
The current draft agreement asks the parties to continue to work together as long as the collaboration is making progress moving therapies towards patients. Continuing to work together does depend on making progress as we move from one stage of research to the next. If the collaboration isn’t working for either partner, each party has the ability to end the collaboration.
Assuming that both parties agree that progress is being made, it is Healx’s intention to continue the work until a therapy reaches patients, and not stop until it is clear that there is no further work that can help the therapy advance. So, if things are moving along at month 24, the work will continue. The 24 month estimate is the typical timeline-some will move more quickly, others more slowly.
We are concerned with information being disclosed when it could be dangerous for patients or when it could jeopardize the path to clinical development and patient access to treatment. In drug repurposing, since the drugs may already be available at the pharmacy, there is always the concern that if the drugs being tested for a new disease indication are known, patients will take or be given the drugs before safety and efficacy can be verified. This is even more important when a proposed therapy includes combinations of drugs that have not been tested together previously. While the success of drug repurposing is higher than the success of traditional drug development, it isn’t 100%. Repurposed drugs should not be prescribed or given to patients outside of a clinical trial or a validated or approved use. Healx is committed to working with our patient group collaborators to make sure the drug predictions remain confidential until safety and efficacy has been established. We know this is difficult for patients and carers who are desperate for a therapy, and we will work relentlessly with our patient group collaborators to shorten the timelines as much as we can, look at compassionate use, early access and other safe and regulated routes to provide patients with access to treatment as quickly as possible.
Second, since the primary goal of the patient group collaborations is to drive therapies to patients, we need to make sure that the drug inventions can be protected and patented to ensure a viable development path to market so that every patient can access the treatment. The patent requirements are very strict, and even accidental sharing of information can invalidate a patent, which can then reduce the likelihood of the therapy getting to patients. It is therefore important that the drug predictions remain confidential.
We would review each of these situations to see what could and could not be shared with other groups, and what the timing on that sharing might be. To ensure patient safety and viable development path to market, we will typically wait until treatments have been validated for safety and efficacy before sharing the information with the patient community. However, if necessary, we can put in place non-disclosure agreements with multiple parties.
We do not expect patient groups to share any data that they do not have the right to share. If an academic researcher or clinician or other entity has data to provide that Healx felt would enhance our ability to predict a treatment for your disease of interest, we would create a data sharing, licence or other type of agreement with that group, individual or entity, giving Healx the rights to use this data. We would hope that the patient group would be helpful in connecting us to those who have useful data to progress treatment development, and support the request for data sharing. However, please do not share with Healx any proprietary data or information that you do not have the right to share.
The IP should be owned by and remain with the inventors. In most cases, then inventors are the Healx drug discovery team and the Healnet platform There may be specific situations in which a patient group might have a relationship with an academic researcher or clinician who has made a discovery that becomes an invention, and in those cases the invention would stay with the academic inventor. The inventor will then typically file the patents to protect the invention and cover the legal costs.
The owner of the data that is provided to the Healnet platform retains ownership. If a patient group, an academic researcher or clinician or other entity have data they want to provide that Healx feels would enhance our ability to generate and validate predictions, we would create a data-sharing or other agreement with that group, individual or entity. Once data is ingested in the Healnet platform it cannot be undone.
All data that comes from the Healnet platform is owned by Healx. There may be instances where it is appropriate and makes sense for Healx to share or license some of this data to the patient group or other entity, and that would be done under a separate agreement. Patient groups would not co-own data generated by Healx.
We’re not setting a limit to the number of partnerships in the long-term but we believe we have sufficient funds to engage with at least 20 patient groups during the next two years of the RTA Programme, during which projects will be staggered to fit everyone’s schedules.
Our funding comes from investors that believe in our mission to use AI/ML to drive rare disease treatments to patients as quickly as possible and at an affordable price. They provide much more than financial support in order to ensure that we can be successful in meeting our mission. We are very grateful to them.
Healx is covering all the costs and is not planning to ask any of the patient groups to make any financial contribution to the prediction and preclinical validation phases of a collaboration. We have the resources to cover the costs of those phases. For all collaborations that reach the clinical stage, we know we will have to raise additional funds to cover the clinical trials and regulatory costs. If a patient group wanted to provide financial resources at that time and enter into a co-development agreement, we are open to the suggestion and willing to explore aor mutually acceptable agreement. We believe we have sufficient funds to engage with at least 20 patient groups during the next two years of the RTA Program.
The actual size of the patient population is not always a relevant factor in deciding whether to apply Healnet to a specific disease. The critical factor is how much data is available about the disease and similar diseases. Healnet has been successful at generating valuable predictions for very rare diseases with a strong scientific and clinical network with good cellular and other preclinical models and published literature.
Almost all reliable disease, drug, patient, medical, cellular, genetic, protein, metabolism and other data are useful in helping Healx make better predictions and do a better job of validation. Healx has accumulated most, if not all, of the publicly available data. Some of this data is reliable, and other data is less so. We spend a lot of time curating the data before it becomes a part of the Healnet platform. We would be eager to be able to utilize additional unpublished data that could improve our predictions. Most information from current/previous research is useful. So is data from screening targets or drugs. If this data is available, we will work with the patient group to create a data-sharing or other appropriate type of agreement so that all parties understand how the data will be used and managed.
Reliable data is generally very useful, and we are looking to our patient group partners to help us find and access whatever useful data is available that they know about. We would have to determine whether we already have this or similar data. If not, we would review how the data is provided and what the value of it would be, to see if it makes sense to add the data to the Healnet platform.
The Healnet platform is designed to find as many drug-disease “hits” as it can. The system ranks the hits according to the algorithms, and also provides the evidence trail so our pharmacologists can then evaluate the hits and their rankings, and select the drugs that make the most sense from a biological standpoint. The more knowledge that is available about a disease, the more robust and successful the predictions should be. If we consider creating combinations of 2 or 3 existing drugs, it is possible that the Healnet system would evaluate about 13 billion possibilities per disease.
We choose the drugs by relying on a combination of our AI/ML Healnet platform and our pharmacology and other human experts. The Healnet platform can use more than 10 different methods to match treatments to diseases. It will provide our pharmacologists with a ranking of the most likely individual drugs that could impact a particular disease. It also provides the evidence it used to make these decisions. Some of these predictions will seem obvious to the pharmacologists, but other predictions will be very novel. The pharmacologists then apply significant scientific and medical knowledge about the disease and the drugs to select the most likely drugs to help a particular disease. Then these individual drugs are tested in preclinical models, and the ones that work the best are often combined to see if they can provide synergistic impact. The best monotherapies and combinations are then tested in the in vivo models, and if they show efficacy, will then be moved to human clinical trials.
There are no limitations and we are not limited by the identified targets. The platform is hypothesis free-it lets the data determine what might be useful, and then the pharmacologists and others decide which of the proposed predictions make the most sense. In diseases that have many unmet needs, we look to our patient group partners to help us prioritise which are most important to try to solve.
We invent novel treatment opportunities by maximizing therapeutic value of safe and already approved treatments, either in monotherapies or combinations. We use drugs that are already approved in at least one jurisdiction. We can also use nutraceuticals (vitamins, minerals, supplements), which are drug-like compounds that have clear biochemical impact in the body, but have never been approved as drugs. The reason that we rely on these drugs and nutraceuticals is that they have, for the most part, been proven safe for use in humans over many years and types of patients, that most are relatively inexpensive and widely available for clinical testing, and together these can reduce the time and the cost of development, so that therapies can reach patients more quickly and at a more reasonable cost.
We choose the drugs by relying on a combination of our AI/ML Healnet platform and our deep pharmacology and drug discovery expertise. The Healnet platform uses more than 10 different computational methods to match drugs to rare diseases. It will provide our pharmacologists with a ranking of the most likely individual drugs that could impact a particular disease. It also provides the evidence it used to make these decisions. Some of these predictions will seem obvious to the pharmacologists, but other predictions will be very novel. The pharmacologists then apply significant scientific and medical knowledge about the disease and the drugs to select the most likely drugs to help a particular disease. Then these individual drugs are tested in preclinical models, and the ones that work the best are often combined to see if they can provide synergistic impact.
The drug testing can be done on cellular and other in vitro, ex vivo and in vivo models. We prefer to test the drugs on validated cell lines from patients, in addition to other cellular validation, and on validated in vivo models that are representative of the disease we are studying. Healx does not have laboratories to conduct these experiments; we work with patient groups to locate the labs that have validated models and we conduct the research there.
So far, we have been able to find good treatment candidates in all of the diseases we have worked on. In the event that we can’t find candidates, we can first determine together if there is additional information that we might access that might allow us to find candidates. If not, we can explore alternative pathways to ensure patient impact.
The RTA Programme is designed to fund all of the work up to, and including the drafting of the clinical trial protocol. If we reach the clinical trial stage successfully, Healx will take the lead to fund and conduct the clinical trials and bring the treatment to patients via a regulatory approval pathway.
We are based in Cambridge, England and spun out of Cambridge University. We do our validation work around the world wherever the best experts are in a particular disease, and where the disease models are the strongest. Our goal is to always secure regulatory approval for the therapies we create, in the US, the EU and other jurisdictions. We will work with the FDA, EMA and others to make sure we have the support and information to have the best chance to secure approval. We are also working with the FDA on global issues concerning the repurposing of drugs to help create new incentives and routes to approval and to label changes for new proven uses.
Healx and our partners want to move therapies to patients as quickly as possible and at an affordable price, so that lives are improved. We have sufficient funding to undertake the prediction and preclinical validation phases of the collaborations, so finances should not have an impact on the timing of these phases. The timing is more reliant on the ability to complete the preclinical development. In some diseases, preclinical validation is quicker, and in some slower, depending on what is necessary to do in the cellular and other models. The same is true for the clinical trials-some will be shorter and some longer. We will cover the clinical costs and lead clinical phase and the regulatory approval.
The opportunity to contribute to the development of a rare disease treatment and be part of a cross-functional partnership, bringing together world-leading experts in artificial intelligence, drug discovery and rare disease. We hope they would believe they were gaining a collaborative partner in Healx willing to utilise our expertise, network and capital to help validate new treatment opportunities. We believe that the researchers, clinicians and other key opinion leaders, in addition to the patients/carers and the patient group, are critical to helping select the right therapies to move to patients and accelerating the path to the clinic. Furthermore, it is likely that the collaboration will result in academic publications.
We would hope that the motivation to a researcher who has unpublished data relevant to a disease or group of diseases, or a drug-disease combination, is that the additional validated data could improve the quality and the speed of validation so that therapies could reach patients more quickly and with greater effect. We are happy to try to create a data licensing or other type of agreement with whomever owns the data so that all parties feel they are receiving value for the information they are providing. If any financial return to the academic institution is to be discussed, this is usually negotiated in the context of the data agreement.
It is possible that those working in a disease area with other companies might see Healx as a competitor, but we hope they don’t. We hope that they will see us as a potential collaborator. Please do not hesitate to introduce us to existing partners, because we have seen that we can achieve more by working together. No organisation can do this alone – we need the patient groups and KOLs to help with disease knowledge, trial design and so many other issues. We need scientists and contrast research organisations (CROs) to help us with preclinical validation. We need sponsors and other funders to help us afford to do this work. We need patients and their carers to participate in trials, and clinicians to carry out the trials. It takes all of us working together to create new treatments.
Yes, not through the RTA programme, which is for patient groups only, but directly with Healx https://healx.io/partnerships/. We are an incredibly collaborative company. By working together with all sorts of partners, including other companies, we will be able to bring more therapies to patients. Company representatives should contact us to explore co-development opportunities. Please contact our Chief Collaboration Officer Dr. Bruce Bloom by clicking here
Yes, not through the RTA Programme, which is for patient groups, but directly with Healx https://healx.io/partnerships/. We are an incredibly collaborative company. By working together with all sorts of partners, including other companies, we will be able to bring more therapies to patients. Company representatives should contact us to explore co-development opportunities. Please contact our Chief Collaboration Officer Dr. Bruce Bloom by clicking here.
Yes, not through the RTA Programme, which is for patient groups, but directly with Healx https://healx.io/partnerships/. We welcome academics and clinicians who have ideas for repurposing for rare diseases, especially those with ideas that are ready for clinical development, to contact us to explore a potential partnership. Please make sure you don’t disclose any confidential information when you contact us initially – once it looks like we should be working together to help patients we can put confidentiality agreements in place to protect your idea. Please contact our Chief Collaboration Officer Dr. Bruce Bloom by clicking here.
Healx works with academics all the time. We want to work with everyone who has knowledge, data, networks, insight, advice, experience that can help us be more successful in our mission to drive therapies to patients. We strive to work with all parties in a way that is fair and equitable. We are able to create these kinds of collaborations in most circumstances
Yes. We welcome contact from anyone who has an idea for repurposing for rare diseases, especially those ideas that are ready for clinical development. Please visit us at https://healx.io/partnerships/ to find out more about our different partnership opportunities. Please make sure you don’t disclose any confidential information when you contact us initially – once it looks like we should be working together to help patients we can put confidentiality agreements in place to protect your idea. Please contact our Chief Collaboration Officer Dr. Bruce Bloom by clicking here.