Target ALS provides support for in vivo proof-of-concept for promising therapeutics in mouse models of ALS. We have partnered with the contract research organization Biospective 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.
All proposals are reviewed by the Target ALS Independent Review Committee.
Key Dates
May 10, 2024
July 2024
August 2024
What We’re Funding
This grant will allow you to evaluate the effect of modulating a candidate therapeutic target on the ALS phenotypes in a TDP-43 mouse model. Biospective has implemented a modified, slower progressing (months) version of the NLS8 (or ΔNLS) model that was originally developed at the University of Pennsylvania (Walker et al., 2015). In the Biospective version, the mice are switched from the Dox diet to a standard diet for a short period and then maintained on a lower Dox protocol developed by Biospective (“Low Dox” model). Phenotypes include cytoplasmic TDP-43 mislocalization, neurodegeneration, and motor deficits. 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 Biospective or with the grantee organization to access these data.
Who Should Apply
- Academic or industry investigators
- No prior or current receipt of funding from Target ALS is required
- All proposals will be evaluated independently of prior or current Target ALS funding
- Applications without demonstrated test article safety (at planned dose) in animal studies will not be considered
- Applicants must have test article in-hand for September 2024 shipment to Biospective
What Our Grants Support
This is an in-kind grant. No funding will be provided directly to the investigator. Target ALS will cover costs of approved projects to be conducted at Biospective. There are several options for study design and readout, with a maximum of 30 mice. Please refer to this slide deck for comprehensive information about study design options.
Studies include:
- Dosing for up to 8 weeks (prophylactic/preventative and interventional dosing are both possible)
- Clinical measures:
- Body weight
- Motor scores
- In vivo Anatomical MRI
- IHC measures:
- Human TDP-43
- pTDP-43 (p409/410)
- GFAP (reactive astrocytes)
- Frozen tissue and terminal fluids may be shipped to investigator
Multiple dosing options are available. Other endpoints may be substituted upon request. Additional experimental groups and endpoints are available at additional cost borne by the investigator.
For more details, please contact us.
