Target ALS | Consortia Developing New Modalities as Therapeutics for ALS

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Event details

Date 18.11.2024
Category Call for proposal
Aim: Target ALS is a medical research foundation committed to the search for effective treatments for Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease. Target ALS funds investigators who collaborate across scientific disciplines and research constituencies to drive medical breakthroughs for the treatment of ALS.

Advances in technology have allowed for some of the first RNA-directed therapies to come to market to treat CNS disorders. Target ALS is seeking to facilitate discovery and development of the next generation of molecules targeting DNA/RNA. The foundation is soliciting proposals from collaborative groups focused on technological innovation of novel modalities in sporadic and familial forms of ALS.

Collaborative consortia working to optimize drug-like properties of small and large molecule therapeutics targeting RNA or DNA for ALS are eligible to apply. Target ALS is particularly interested in supporting projects that propose the study of:
  • New antisense oligonucleotide chemistry/design, siRNA/disiRNA, CRiSPR, Zinc Finger, and similar technological approaches targeting genetic mutations associated with ALS
  • Enhanced brain delivery using conjugation strategies, lipid nanoparticles, novel capsids, BBB-penetrant AAV, or similar platforms
  • Molecules having demonstrated cell-based activity but needing additional in vivo validation of effective delivery to brain and spinal cord, pharmacodynamic activity on the target of interest, and tolerability
  • The novel modality must be applied to a genetic target associated with ALS
Funding: Max $400,000/year (max. $200,000 per lab per year)

Duration: 24 months (possible 12 months in addition based on progress)

Eligibility: This call is focused on bringing academic investigators together with other academics or industry partners to work collaboratively on developing the next generation of gene therapies. Only collaborative projects will be considered.
  • Collaborative projects for this call may comprise groups of 2-4 laboratories synergizing to develop the molecule of interest 
  • One industry partner (CRO, biotech, pharma) is recommended
  • All collaborators must provide a copy of the executed agreement between their institutions on data/IP sharing OR provide a document that confirms each Technology Transfer Office has been made aware of the collaborative project and the need to sign a data/IP sharing agreement as part of the full proposal submission. NOTE: A fully executed data/IP sharing agreement must be shared within 60 days of notification of award or the grant will be rescinded.
  • A Principal Investigator can only participate in one LOI submission.
  • Principal Investigators who have two or more active competitive grants from Target ALS are not eligible to apply. 
How to Apply: The two-step application process begins with a Letter of Intent. The letter of intent can be filled in directly on the application portal. Selected applicants will be notified in January 2025 and invited to submit a full proposal. At the full proposal stage, a detailed budget is required. Budgets can include researcher salaries and fringe benefits, materials and consumables, equipment, and overhead of 15%.

Deadline, Letter of Intent: 18 November 2024
Deadline, Full Proposal: 21 February 2024

Further information
  • More information about the program is available here
  • The application portal can be found here
  • For any other questions, please contact the Research Office

Practical information

  • General public
  • Free

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