Target ALS – In Vivo Target Validation
Target ALS and the ALS Association provide support for in vivo proof-of-concept for promising therapeutics in mouse models of ALS. We have partnered with The Jackson Laboratory (JAX) to enable evaluation of novel therapeutics for target engagement and disease-modifying potential in ALS. Test articles – small molecules, biologics, gene therapy, or antisense oligonucleotides – are provided by the investigator. Results from these studies have the potential to catapult ALS drug candidates from preclinical to clinical pipelines, accelerating development of novel therapeutics.
This in-kind grant will allow you to evaluate the effect of modulating a candidate therapeutic target on the ALS phenotypes in a standardized AAV9-based 149 G4C2 repeat C9orf72 mouse model (Chew et al., 2019). Please refer to this slide deck for a complete understanding of the model, phenotypes, and readouts.
Confidentiality of investigator’s data, research, and intellectual property will be strictly honored. Target ALS does not seek ownership of any intellectual property or financial gains that result because of its funding. All data generated from studies will be shared with Target ALS for internal quality control purposes. Target ALS will not enter into any confidentiality agreements with JAX or with the grantee organization to access these data. Data generated at JAX is covered by the confidentiality clause of the General Terms and Conditions (General terms and conditions (jax.org))
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Website: https://www.targetals.org/in-vivo-target-validation/