This grant provides targeted funding to drive the discovery, development, or translation of a gene therapy for ADSL Deficiency (ADSLD). We aim to support innovative, collaborative research that advances promising ideas from the laboratory toward treatments.

​This rapid-response grant will fill critical funding gaps in new or ongoing investigations, and support projects that can generate the preliminary data needed to attract larger governmental, foundation, or industry investments.

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Projects eligible for funding include:​

1. Gene Therapy

Vector optimization, Delivery strategies, Dose optimization, Preclinical efficacy studies, Manufacturing readiness

Applicants must be affiliated with an eligible institution, including:

• Universities

• Medical schools

• Hospitals

• Nonprofit research institutions

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The Principal Investigator must demonstrate appropriate expertise and institutional support for the proposed project.

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Collaborative, multidisciplinary applications are encouraged, and subject to the Foundation's legal, financial, and administrative ability to make the award.

Grant funds may be used for reasonable and necessary costs directly related to the approved research project, including:

• Research personnel

• Laboratory supplies and reagents

• Animal model studies

• Cell culture and patient-derived model development

• Specialized testing or core facility services

• Data analysis

• Equipment directly required for the project

• Project-specific research expenses

• Reasonable publication or data-sharing cost

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Indirect costs must comprise no more than 10% of total direct costs.