Gene therapy has emerged as a promising medical approach, offering a potential treatment path for diseases that have long been considered incurable. This therapy involves the manipulation of genetic material within a patient's cells to alleviate or cure diseases.
Despite the significant progress made in this field, there remain numerous disorders for which effective gene therapies have yet to be developed. Many challenges arise either from the complexity of the diseases, as some involve multiple genes, or from delivery issues, as getting therapeutic genes into the right cells safely and efficiently remains a major hurdle. For instance, certain tissues, like the brain or heart, are difficult to reach, and some gene delivery systems, such as viral vectors, can trigger immune responses or have limited targeting capabilities. Furthermore, the identification and validation of novel targets for gene therapy pose significant challenges.
Discovering suitable targets, especially for common diseases, requires a deep understanding of how specific genes contribute to disease, which is often complicated by incomplete knowledge of genetic functions. Even when promising targets are identified, laboratory models do not always predict unintended side effects or how these therapies will behave in humans, making it difficult to ensure both safety and efficacy. Therefore, there is an urgent need to explore and develop new gene therapies to address these unmet medical needs.
We are looking for innovative research projects that can identify and explore novel targets for gene therapy, particularly in CNS (central nervous system) and CVM (cardiovascular and metabolism) related diseases. We are interested in proposals that explore therapeutic effects through gene supplementation, knockdown, or a combination of both. The targets should be delivered by Adeno-Associated Virus (AAV) to the relevant tissues and cell types and should be distinguishable from existing therapies.
At Daiichi Sankyo, we attach significant importance to working with academic institutions, startups and bioventure companies to discover new therapeutics in the place where hypotheses are brought and tested in order to expand possibilities for scientific innovation breakthrough. We build sustainable relationships with partner institutions and companies through open and fair alliance management and trust based on mutual respect as the foundation for effective collaborations. Our goal is to jointly create new value for patients by maximizing each other’s expertise and strengths.
https://www.daiichisankyo.com/rd/strategy_operations/open_innovation/
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