Program Details & FAQs

Open Aperture — Program Details & Application

This page summarizes the application process, what information to prepare, and what the standard MTA permits. When you are ready, start the application at the bottom of the page.

How the program works

  1. Complete and submit your application. Then, Apertura Gene Therapy will log your submission and begin the review process.
  2. Our team will check for eligibility and completeness. If something is missing, we will ask you to update it; if not eligible, Apertura Gene Therapy will reach out to discuss alternative access options.
  3. Once eligibility is confirmed, Apertura Gene Therapy will provide your Technology Transfer Office (TTO) with our MTA package, which includes a cover letter, a CDA agreement, and the MTA agreement.
  4. After execution of the agreements, Apertura Gene Therapy will introduce you to vendor contacts and share our TfR1 CapX package that will include useful information related to manufacturing and pre-clinical assessments using TfR1 CapX.

Information you will need to provide to apply

Applicant & PI
  • Applicant name and email
  • Academic institution
  • Role
  • Principal Investigator’s name and email
Technology Transfer Office
  • TTO contact name and email
For each indication or program:
  • Disease indication
  • Capsids used to date
  • A brief description of the current development stage
  • A brief description of the development plan & intended use of TfR1 CapX
  • Next anticipated regulatory meeting type and date

Eligibility

  • Institution type: Accredited academic research entity.
  • Use case: Non-commercial research only. Not for clinical administration.
  • PI/TTO participation: Named PI and valid TTO contact required.
  • Compliance: Agreement to standard MTA terms and applicable regulations.

Final eligibility is determined during internal review.

What the standard MTA allows / does not allow

Allows:

  • Use of TfR1 CapX materials for academic, non-commercial research.
  • Internal evaluation in the PI’s lab and their contracted parties, including CROs and CDMOs, if approved.
  • Publishing of data using TfR1 CapX is allowed; However, it is subject to the MTA’s restrictions on when data is published – Only permitted 18-months after execution of the agreement or the Broad Institute’s publication on TfR1 CapX, whichever occurs first​
    • This is to protect our scientific founder and ensure their publication on TfR1 CapX is the first​
    • The 18-month restriction is unlikely to impact publishing timelines as study design, execution, analysis, and manuscript authoring will likely take longer than 18-months
  • Limited data sharing for academic purposes under confidentiality obligations.

Does not allow:

  • Clinical use, diagnostic use in humans, or manufacturing for human administration.
  • Commercial use, sublicensing, or redistribution to third parties.
  • Reverse engineering or sequence disclosure beyond what the MTA permits.
  • Use outside the approved indication(s) or labs named in the agreement.
  • Use of TfR1 CapX in regulatory submissions without prior notification to Apertura Gene Therapy.

After signature: what you will receive

  • Preclinical therapeutic development roadmap when using TfR1 CapX.
  • CMC documentation package that includes resources to jumpstart production and analytics of TfR1 CapX products.
  • Introductions to relevant vendor contacts with experience working with TfR1 CapX therapeutics for logistical support.

Frequently Asked Questions

Who can apply?
PIs and research teams at accredited academic institutions conducting non-commercial research.

Is there a cost?
The program provides no-cost access to TfR1 CapX for eligible academic researchers.

What if my institution already has an MTA with Apertura Gene Therapy?
Apertura Gene Therapy will add your lab as an exhibit under the existing agreement, after review.

What happens if my application is incomplete or not eligible?
Apertura Gene Therapy will ask you to complete missing items or, if not eligible, discuss alternative access options.

Can I include multiple indications in one application?
Yes. Up to five indications or programs per submission, each with a brief development summary.

Can additional indications be added after the MTA is completed?
Yes. Additional indications of TfR1 CapX by the PI or other PIs/Labs at the institution can be added at any time.

When will we hear back?
Timing depends on your institution’s review process. Apertura Gene Therapy will move quickly to ensure access is provided as soon as possible.

Can we publish results?
Generally yes, subject to the MTA’s publication terms (e.g., notice period, confidentiality).

Is there a path to take a therapeutic product using TfR1 CapX to the clinic?
Yes. Apertura Gene Therapy wants to support patients that could benefit from gene therapies using TfR1 CapX. If you have promising results that you believe should advance to the clinic, contact Apertura Gene Therapy to discuss a commercial license.

This page summarizes the Open Aperture program. Final terms are defined by the executed agreement between your institution and Apertura.

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