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.
Ready to start the application process?
***Attention applicants – we have a new application portal. Learn more.
Key Dates
March 24, 2025
June 2025
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 rNLS8 (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 must demonstrate test article safety (at planned dose) in animal studies and must include brain penetrance data to be considered
- Applicants must have test article in-hand for July 2025 for 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 45 mice. Please refer to this slide deck for comprehensive information about study design options.
Funded groups must enter into a standard, Target ALS-approved, non-negotiable Material Transfer Agreement (MTA) with Biospective. A copy of this MTA can be provided to applicants upon request.
Study endpoints may include:
- Dosing for up to 8 weeks (prophylactic/preventative and interventional dosing are both possible)
- Clinical measures:
- Body weight
- Motor scores (including grip strength)
- In vivo Anatomical MRI
- Longitudinal CT imaging of hindlimb muscle atrophy
- Gastrocnemius muscle electrophysiology (CMAP)
- 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.
Funded studies to be initiated (with staggered study starts) in Fall 2025, with in vivo work expected to end December 2025 for all studies
For more details, please contact us.
New Target ALS Application Portal
We are in the process of changing our online grant application portal.
- If you did not apply to the Target ALS New Therapeutic Modalities Consortia funding call, you will need to create a new user account, called a Blackbaud ID, even if you have applied to our grants in the past.
- You will be able to share your applications with other key stakeholders in your organization.
- If you have a current Target ALS grant, you will not see it in this new application portal (we’re working on unifying them).
- Add noreply@yourcause.com to your safe senders list to make sure you receive our emails and notifications.
Ready to start the application process?
