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Exploratory research for novel gene therapy targets
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Get them answered by the team at Daiichi Sankyo.
Background

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.

What we're looking for

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.

Solutions of interest include:
  • Investigation of novel disease mechanisms and identification of gene targets that are not adequately addressed by existing therapies, focusing on approaches to target protein overexpression or harmful mutations using AAV-delivered RNA interference (RNAi) or CRISPR-based gene knockdown.
  • In vitro models using human tissues or patient-derived cells for omics and genomic analysis that can evaluate pharmacological effects and validate selectivity of the knockdown.
  • Exploratory research focusing on novel gene therapy targets with high clinical translatability, leveraging omics or genomic analysis from patient-derived tissues and cells to correlate target gene modulation with disease phenotypes.
  • Use of novel CRISPR variants or alternative gene-editing technologies that limit off-target activity while delivering therapeutic effects via AAV.
  • Research that can evaluate the pharmacological effects mediated by novel targets in in vitro/in vivo systems.
  • AAV-delivered gene therapies that produce proteins, enzymes, or other therapeutic agents secreted into systemic circulation, addressing diseases that require widespread effects (e.g., lysosomal storage disorders), along with in vitro and in vivo models to monitor secretion levels and assess efficacy and safety.
Our must-have requirements are:
  • The target must exhibit therapeutic effects in tissues and cell types that are accessible through AAV delivery, such as liver, muscle, or the central nervous system. This includes cases where the therapeutic effect is achieved by the drug being secreted into systemic circulation.
  • For a novel target of single gene knockdown, the target should be limited to those that can be differentiated by gene therapy from existing therapies against the same target.
Our nice-to-have's are:
  • An exploratory study of novel targets with clinical translatability using patient cells and tissues that allow for omics data analysis or genomic analysis.
  • Genome editing technologies that offer higher selectivity for target sequences, ensuring high efficacy and safety.
What's out of scope:
  • Diseases with a prevalence of less than 1 in 100,000 individuals in Japan and the US.
  • Therapies aimed at treating cancer.
Acceptable technology readiness levels (TRL):
Levels 1-5
What we can offer you
Eligible partnership models:
Sponsored research
Benefits:
Sponsored Research
Funding is proposal dependent, with up to $ 100K for 12-month project with potential follow-on funding for 1 year.
Who we are

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/

 Learn more
Reviewers
RS
Ryusuke Sugita
Senior Researcher
MN
Masatoshi Nagamochi
Associate Director
MK
Mikio Kato
senior director
KS
Kousei Shimada
Director
Q&A with Daiichi Sankyo

Ask the team at Daiichi Sankyo any questions you have about this RFP.

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Q.
Would you support, through in-kind funding, whole-genome long-read sequencing and validation to discover novel variants associated with specific cardiovascular diseases
2
A.
Thank you for your question. If the prevalence of the "specific heart disease" meets our requirements and there is technical novelty or uniqueness in the sequencing, we would be interested. Otherwise, it would fall outside our scope. We think that discussions regarding the technical possibilities and costs of sequencing will be necessary depending on the proposal details. Best regards, Ryusuke
RS
Ryusuke Sugita, Senior Researcher, Daiichi Sankyo
October 31, 2024
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0
Q.
Would APOE4 for AD be of interest?
1
A.
Thank you for your question and sorry for the late reply. APOE4 is a well-known risk factor for AD and may be a potential therapeutic target for AD. Several pharmaceutical companies are currently engaged in research and development targeting APOE. Therefore, while we would be interested if there is novelty in the drug discovery approach, it would fall outside our scope if it lacks such innovation. Best regards, Ryusuke
RS
Ryusuke Sugita, Senior Researcher, Daiichi Sankyo
October 28, 2024
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0
Q.
would gene GLA/Fabry disease be of interest?
1
A.
Thank you for your question. While the prevalence of the disease meets our criteria, GLA/Fabry disease is a well-known target in the gene therapy field, and thus the aspect of novelty may not align with our scope. It is desirable that the proposed therapeutic concept has novelty and a clear advantage over existing products, including those currently in clinical trials. Best regards, Ryusuke
RS
Ryusuke Sugita, Senior Researcher, Daiichi Sankyo
October 15, 2024
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Deadline: November 30
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