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.
All proposals are reviewed by the Target ALS Independent Review Committee.
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Key Dates
What We’re Funding
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))
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 November 2024 shipment to JAX
What our Grant Supports
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 JAX. Cost sharing (extra readouts and/or mice paid by grantee) is not permitted for this funding call. Costs of shipping samples at the end of the study will go to the grantee.
Mice will be injected at birth with AAV9 encoding 149 G4C2 repeats and the study readouts performed at 6 or 9 months of age. Please refer to this slide deck for comprehensive information about study design options, looking carefully at the phenotypic characteristics at your desired time of study termination.
You may select from three of the following readouts:
- Sense and anti-sense foci
- Poly (GP) Immunoassay
- Immunohistochemistry quantification of:
- pTDP-43 (offered only for studies aging mice to 9 months)
- poly(GA) IHC
- poly(GR) IHC
- GFAP
- Iba1
Behavioral tests
- Open field test
- Contextual Fear Conditioning*
*JAX has conducted contextual and cued fear conditioning. AAV9-(G4C2)149 mice have a significant learning and memory deficit as measured by contextual fear conditioning. There is no deficit in the cued fear conditioning task.
Sample study design, other options are possible with a maximum of 30 mice with (G4C2)149 AAV.
Ready to start the application process?
Questions? Don’t hesitate to contact us.
