FAQs: Retinal Gene Therapy
WHAT IS GENE REPLACEMENT THERAPY?
Gene Replacement Therapy is a cutting-edge medical treatment that involves replacing a defective gene within a patient's cells with a healthy copy to correct genetic disorders. First conceptualized in the 1970s, it gained momentum with the successful treatment of several severe conditions in the 2000s. The therapy works by using vectors, typically modified viruses, to deliver the healthy gene to the patient's cells, allowing the cells to produce the correct proteins needed for normal function.
GENE REPLACEMENT & THE RETINA
One notable success is the treatment of Leber's congenital amaurosis (LCA), a rare inherited eye disorder, where gene therapy restored vision in patients who were previously blind due to a mutation in the gene RPE65.
The retina is considered a highly suitable location for gene therapy delivery because it is easy to access via basic surgery or injection, and the blood-brain barrier minimizes immune responses that can be caused by injecting a virus (vector) into the body.
HOW LONG DOES A CUSTOM GENE THERAPY TAKE TO MAKE? HOW MUCH DOES IT COST?
Based on previous examples of custom gene replacement therapies developed by other foundations, we expect it to take ~4 years to reach our first patient. Our goal is to deliver a dose of the therapy in 2028.
The total cost of the project is roughly $2.5-3M dollars; however, this cost can be significantly reduced as key foundational technologies currently under development become more available. Further, while the Ringel Family and Kizuna Foundation expect to fundraise a significant portion of this funding, there are numerous grants that will help fund key parts of the development plan such as manufacturing when we reach this stage. The key is to get started to have a program “in the game”
HOW LIKELY IS IT TO WORK?
Ultimately, we cannot be certain if this approach will bring therapeutic benefit to Stevie, Natalie, or other KIZ patients. Based on the size and function of the KIZ gene, we have a high degree of confidence that we can successfully deliver a healthy copy of the gene to the right retinal cells. How this translates to vision preservation is unknown, and this is a key reason we need support - only rigorous testing can make us confident this treatment is worth injecting into the retina.
Our action plan
We have a fundraising goal of $400K for 2025, and through January 2025 we have ~$260K remaining to raise.
We are raising this money to create, characterize, and demonstrate initial scientific validity of our therapy. Your support means we can move quickly through these stages*
*Note: Dollar amounts are estimates and subject to change
Our Funding Policies
Kizuna Foundation is committed to operating efficiently with the maximum amount of donated funds directly impacting patient treatment development.
As of October 2025, Kizuna Foundation’s executive leadership has enacted a policy strictly to not pay overhead, administrative, processing, or other G&A fees when granting research dollars to partners.
Full IDC Policy
Scope. Applies to all grants, contracts, subawards, and payments (“Awards”) issued by Kizuna Foundation to any recipient organization, including universities (e.g., University of Florida), hospitals, non-profits, and companies.
Definitions.
Indirect Costs / Overhead / Facilities & Administrative (F&A) include, without limitation: university or departmental overhead; facilities, administrative, processing, compliance, sponsored programs, or post-award fees; general office administration; utilities; building depreciation; network/IT infrastructure; administrative personnel not working exclusively on the Award; and any percentage-based fee (F&A/IDC) applied to a cost base (MTDC, TDC, or otherwise).
Direct Costs are costs that are specifically and solely attributable to the Award’s approved scope of work and can be documented to the project with reasonable accuracy.
Policy.
No IDC Allowed. Kizuna Foundation will not pay indirect, overhead, administrative, processing, or other G&A fees. The IDC rate is capped at 0% on any cost base.
No Relabeling. Indirects may not be reclassified as “administrative supplements,” “compliance fees,” “institutional support,” “facility use,” “proposal processing,” “grant management,” “export control,” “IT/security,” “hazardous waste,” or similar. Any such charges will be removed.
Gifts vs. Grants. This policy applies whether the Award is issued as a grant, contract, subaward, clinical study agreement, service agreement, or gift. (Institutions may not convert IDC into gift assessment fees or similar charges.)
Subrecipients/Consultants. This policy flows down: subawards and consultants under the Award may not charge IDC to Kizuna funds.
Budget Format. Recipients must provide a line-item budget that clearly distinguishes Direct Costs from any institutional fees.
Acceptance. Receipt or use of funds constitutes acceptance of this policy and supersedes recipient institutional IDC policies for this Award.
Allowable Direct Costs (examples).
Personnel directly performing the project: PI/Co-I effort, research staff, students; associated fringe benefits at auditable, institution-standard rates.
Project-specific supplies and reagents, animals and per-diems, disposables, cell lines, sequencing/library prep, and core facility recharge rates at published, non-discriminatory user fees.
Project-specific equipment (≥ useful life 1+ year) when justified; maintenance if uniquely required for the project.
Participant/subject costs and stipends; clinical visit costs billed at standard rates.
Necessary compliance pass-throughs tied to the project (e.g., external IRB fees, IACUC per-protocol fees if itemized and project-specific), biosafety approvals, and research-related shipping/customs.
Data acquisition/analysis services uniquely required for the project; reasonable publication and open-access fees arising from the Award.
Travel essential to executing the project (not general conferences unless tied to project milestones).
Tuition/fees only if the student is key project personnel, effort is budgeted, and the institution cannot waive; otherwise not allowed.
Unallowable Costs (non-exhaustive).
Institutional F&A/IDC, departmental or college taxes; sponsored programs office fees; proposal processing or post-award fees; gift/assessment taxes; network/IT backbone charges; facilities depreciation; building ops; general admin staff not exclusively assigned to the project; patent prosecution unrelated to Kizuna-funded IP; general liability/insurance pools; general office supplies/equipment; memberships; unrelated conferences; entertainment.
Exceptions.
Exceptions are rare and require prior written approval by Kizuna’s CFO/CEO before award signature. Any approved exception must be stated as a fixed dollar amount (not a percentage) on a named line item.
Compliance & Audit.
Kizuna may request documentation supporting direct charges and may disallow costs that do not meet this policy. Disallowed costs must be removed or refunded within 30 days.
Material non-compliance may lead to funding hold or termination.
Effective Date.
Effective for all Awards issued on or after October 1st, 2025; applies to amendments and additional funding to prior Awards unless expressly exempted in writing.