DREAMS plans to pioneer advances in the ability to find drugs for serious diseases using Artificial Intelligence (AI) and induced pluripotent stem cells to increase the possibilities, reduce costs, decrease the time needed and expand the range of diseases that can be treated with a single drug.
Furthermore, DREAMS aims to develop the first AI and iPSC-based drug discovery platform to
+ Find treatments for rare NMDs.
+ Computationally design drugs that are, from the onset, engineered to target multiple diseases.
+ Be used to solve a wide breadth of drug discovery challenges in a single project, including target discovery, drug design, indication discovery, etc.
This new platform will be conceived to be reused in the future so DREAMS can lead to the discovery of treatments for rare NMDs, far beyond our initial 5 indications of interest.
The creation of cellular assays of our starting diseases and the generation of data through these assays.
The discovery and validation of drugs and extended therapeutic indications.
The preparation of clinical validation of our treatments.
DREAMS will start from a subset of 5 rare orphan neuromuscular disorders with a known genetic origin, characterised molecular mechanisms, and existing biomarkers. These disorders have been selected because they share common pathophysiological characteristics related to dysfunctions of autophagy:
The two pharmacological approaches developed in DREAMS will collectively lead to breakthrough conceptual and scientific proof-of- concept studies on NMD that could be reusable for any other group of diseases sharing pathological features.
These innovations will pave the way for the future development of new effective pharmacological therapies proposing an all-in-one innovative methodology to treat multiple RDs.
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DREAMS’ first approach involves a “Forward pharmacology”, which is planned to modify existing tools currently used for personalised medicine of rare diseases and extend their therapeutic potential by combining them with AI based predictions. More specifically, AI will firstly enable to predict commonalities of a subset of diseases of interest, and secondly to discover and design safe and effective drug candidates that specifically act on those commonalities.
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In parallel, a second approach will be developed in DREAMS, called “Reverse pharmacology”. This approach aims to take advantage of the unique dataset generated throughout the project to develop an innovative “reverse” pharmacological approach, where additional rare diseases, that could be treated by the most promising drugs candidates, will be discovered through AI-based methodologies.
DREAMS plans to pioneer advances in the ability to find drugs for serious diseases using Artificial Intelligence (AI) and computational techniques to increase the possibilities, reduce costs, decrease the time needed and expand the range of diseases that can be treated with a single drug.
Furthermore, DREAMS aims to develop the first AI-based drug discovery platform to
+ Find treatments for rare NMDs.
+ Computationally design drugs that are, from the onset, engineered to target multiple diseases.
+ Be used to solve a wide breadth of drug discovery challenges in a single project, including target discovery, drug design, indication discovery, etc.
In addition, DREAMS will generate a unique reusable iPSC/AI powered platform to find treatments for rare NMDs that will revolutionise this area of research.
Finally, the project aims to innovate by designing an adaptive clinical trial with the active participation of patients and regulatory experts to facilitate the implementation of clinical trials for a small and heterogeneous group of RD patients.
Beyond the scientific improvement of the state of the art and the identification of potential therapies, this project will also methodologically innovate by integrating patients and clinicians from the earliest stages of the preclinical and clinical development to involve all needed actors to solve the current limitations in RDs.
