US FDA Grants Expanded Approval of ELEVIDYS Gene Therapy for DMD Patients Ages 4 and Above

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MDA Kickstart Program

*This is a new program

Gene therapy delivered via adeno-associated viruses (AAVs) has proven to be a feasible strategy to treat the underlying genetic cause of numerous neuromuscular diseases (NMDs). Accordingly, many investigators have pursued AAV-based gene replacement strategies to correct various forms of NMDs, with in vitro and in vivo proof-of-concept data at hand and ready for the next step towards clinical translation. While there is expanding interest from industry to support translation studies for AAV gene therapies, these have been limited to more common NMD disease subtypes, with minimal commercial interest for the ultra-rare indications. As a strategy to lower commercial barriers and de-risk development of gene therapies for ultra-rare neurological diseases, the NIH has created funding and resource support via the Ultra-rare Gene-based Therapy Network (URGenT) program. The entry requirements for NIH’s URGenT program is largely targeted toward IND-enabling studies. MDA recognizes that investigators may not have access to personnel with the required expertise or resources required to reach earlier stages such as assembly of a pre-IND package. As a strategy to address the pressing need to lower barriers for ultra-rare gene therapies, MDA is introducing the Kickstart Program to support selected gene therapy programs reach the pre-IND stage of the clinical translation process. Successful grant applicant(s) of the Kickstart Program will have access to resources such as contract research organizations (CROs) and personnel support required for pre-IND package development. MDA’s Kickstart Program will put together a collaborative team consisting of in-house and external experts, and the applicant, to drive the chosen project(s) towards a complete pre-IND package, thus moving the program into position for follow-on grant funding. Examples for the type of activities supported by MDA’s Kickstart Program can be found in Table 1.

Table 1: Types of support available via Kickstart Program.

Types of pre-IND-enabling support
Personnel Specialized ad-hoc consultants: project manager, project lead, regulatory expert, clinical consultants
Research support
  • Research or Good Laboratory Practice (GLP) grade AAV manufacturing and product quality
  • Chemistry, Manufacturing and Controls (CMC) activities
  • Dose-escalation and biodistribution preclinical studies
  • Experimental (Animal) pharmacology / toxicology study
  • Potency assay development
  • Extended Proof-of-Concept (POC) studies
  • Development of regulatory strategy and support for FDA filings
  • Development of Quality Assurance strategy
  • Medical chart review or other plan to acquire natural history data

Scope and eligibility:

The applicant must propose a gene therapy strategy to target an underlying genetic defect of an ultra-rare neuromuscular disease condition affecting less than 1000 people in the United States for which there is no disease-modifying therapy available. For our pilot Kickstart project, we will limit gene correction strategy to gene replacement via AAV. Eligible applicants include principal investigators at academic institutes, non-profit organizations with 501(c)(3) designations and therapeutic development companies. Applicants are restricted to the United States for compliance with FDA regulatory guidelines for gene therapy. Preference will be given to applications that have:

  • Completion of gene therapy construct design
  • Proof-of-concept demonstration using in vitro or in vivo models
  • High likelihood of IND-approval and/or clinical translation within two years

LOIs should address the following points:

  • Background of genetic disease to be addressed
  • Experimental work completed to date
  • Details of the personnel/team applying
  • Goals of the Kickstart collaboration with MDA

Kickstart FAQ:

  1. Do I need to have extensive data in hand to apply for Kickstart? No, the unmet need and potential feasibility of the project are most important. Kickstart is designed to help make such projects competitive for follow-on funding or for investment funding.
  2. If I am selected for the Kickstart program do I receive funds directly? Decisions will be made on a case by case basis. You will work with the MDA Kickstart team to determine what services are needed, many of which may be paid directly by Kickstart to CROs or consultants. If more extensive POC data is required we may fund your lab/company directly to do this work in collaboration with the MDA Kickstart team.
  3. Do I need to own/have access to IP to be considered for Kickstart? Yes. If you don’t have the required IP in hand Kickstart may be able to work with you to secure the necessary IP. Follow-up with Kickstart staff if you have questions.
  4. I represent a biotech company. Can we apply for the Kickstart program? Yes. If you have something in your portfolio that meets Kickstart criteria you may apply to the program. If you are accepted, either the project will be co-developed with Kickstart and/or Kickstart may request right of first refusal to in-license the project on favorable terms.
  5. What if I have already had a pre-IND meeting—can I still apply to Kickstart? Yes, but we will want to understand why you have not applied for the NINDS URGenT program.
  6. If I have applied to NINDS URGenT program or for other similar funding sources and been turned down can I still apply to Kickstart? Yes. If you project is deemed to have potential we can help you overcome critiques to make a stronger application to those other fundings sources.
  7. How long does the Kickstart program last? We will design a program that is 1-2 years in duration and designed to meet a particular inflection point, such as the submission of a pre-IND application.
  8. Can multiple partners apply for Kickstart? Yes. In that case we will put together a memorandum of understanding or contract, if needed. to describe expectations for all partners. For example, this could be a family with an academic investigator or an academic investigator working with a company or any of these in collaboration with another nonprofit organization
  9. We aren’t academic researchers or part of a biotechnology company but our family is affected by this disease—can we apply for the Kickstart program? Yes. Ideally you will have some academic collaborators and other resources identified to be successful in your application.
  10. How does Kickstart operate in practice? If your project is selected for Kickstart we will assemble a core project team consisting of MDA Kickstart staff and 2-3 people that you identify. We will create a milestone-driven project plan and budget needed to move the project to the next inflection point (generally a pre-IND meeting). We will identify the workstreams required, contract with CROs and consultants as needed and then meet weekly as a project team to move the work forward.
  11. What experience does MDA have in gene therapy drug development?
    1. The Kickstart team will be headed by MDA Chief Research Officer, Sharon Hesterlee, who has experience as a project lead for three gene therapy programs at Pfizer, as CEO of Askbio spin-out Lion Therapeutics, where she oversaw the development of Askbio’s LGMD2I gene therapy program and as Executive Vice President and Head of Portfolio for Askbio. In addition she has over 20 years of experience in the neuromuscular space.
    2. Maryna Kolachavina will serve as Kickstart’s primary Project Coordinator. Maryna has over 17 years experience in biopharmaceutical drug development and has managed over 169 rare and orphan projects in 40 therapeutic classes. She has published extensively in the space of rare and orphan drugs.
    3. MDA VP for Research Angela Lek will serve as the primary MDA liaison for Kickstart. Angela has extensive experience in neuromuscular disease pathophysiology and personal experience in gene therapy development
  12. What if I’m not happy with some aspect of the Kickstart process or would like to withdraw for any reason? What is my recourse? The project agreement will describe what happens if Kickstart misses pre-determined milestones or if either side breaches other aspects of the agreement and what actions are available in those situations.
  13. Who makes decisions about project direction in Kickstart? Decisions will be made collaboratively but the Kickstart team has ultimate control over the direction of the project.
  14. What reporting and communication should I expect to receive from the Kickstart team? You will be part of a weekly core project team meeting and you will receive a copy of the official project plan and milestones and written reports on achievement of or progress toward achieving milestones.
  15. What can I take with me at the conclusion of a Kickstart Program? This will depend on what is pre-determined in the project agreement. Kickstart will generally require some return on its investment, whether this is partial or total ownership of IP, right of first refusal, future royalty rights etc. Kickstart can be very flexible in how this ROI is accomplished.
  16. What if my Kickstart program meets all of its milestones successfully but fails to obtain follow-on grant or investor funding? In this case Kickstart may consider taking on the project for a phase II further round of de-risking. This may depend on whether criticisms from funding agencies or investors are addressable with further de-risking.
  17. How are projects are selected? Projects will be selected by MDA’s Kickstart team based on unmet need, technical feasibility, and project team compatibility. Commercial potential will not be a major factor in selection.
  18. Does Kickstart have an external advisory committee? Yes, this is being established and will consist of experts in various aspects of gene therapy drug development including manufacturing and clinical operations.

For questions regarding MDA's Kickstart Program, please contact

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