Gene Therapy: A Step Toward Clinical Trials

The drug/therapy approval process in the US is a lengthy and complex procedure that involves several stages.

The first stage is drug discovery, where a potential drug candidate is identified through laboratory research and testing. Before filing an Investigational New Drug (IND) application, a pre-IND meeting is held with the Food and Drug Administration (FDA) to discuss the proposed clinical trial design, safety considerations, and other relevant aspects. This is the stage we are currently fundraising for.

We are fundraising to prepare the material for the meeting with the FDA, we need your support.

The next stage is preclinical development, where the drug candidate undergoes animal testing to determine its safety and efficacy. The IND application is submitted to the FDA at this stage, including data from the preclinical studies and the proposed clinical trial protocol.

Once the IND is approved, the drug candidate can enter clinical development, which is divided into three phases. Phase 1 trials involve a small group of healthy volunteers to assess safety, dosage, and potential side effects. Phase 2 trials involve a larger group of patients with the condition the drug is targeting, to determine efficacy and further assess safety. Phase 3 trials involve an even larger group of patients to confirm efficacy and monitor adverse effects.

After successful completion of the clinical trials, the drug developer submits a New Drug Application (NDA) to the FDA for review and approval. The FDA reviews the data from the clinical trials, as well as information on the manufacturing, labeling, and distribution of the drug, before making a decision on approval.

Finally, post-marketing surveillance is conducted to monitor the long-term safety and effectiveness of the drug once it is on the market. This stage includes monitoring adverse events, tracking patient outcomes, and conducting additional studies as necessary.

What does the Pre-IND Meeting entail?

Thanks to your support and the money donated, we have completed the following very important steps:

Research and development: Conduct extensive research on gene therapy constructs candidate for NF2, to restore Merlin production.  Several AAV constructs were created as well as NF2 cell lines.

Preclinical studies: Perform preclinical studies in vitro and in vivo to evaluate the construct candidate’s safety, and efficacy. This data will support our pre-IND meeting request and subsequent IND application.

Now with your support, we are fundraising to achieve the following steps (Pre-IND Phase):

1. Prepare the pre-IND meeting package: Compile all relevant data and information on our therapy candidate, including a summary of the research conducted, preclinical study results, the proposed clinical trial design, and any other information that may help the FDA understand our drug candidate and its intended use. This package will be submitted to the FDA as part of our pre-IND meeting request.

2. Submit a pre-IND meeting request: Draft and submit a formal meeting request to the FDA, which should include the meeting package mentioned in step 3, along with a list of specific questions for the FDA to address during the meeting. Clearly outline the drug development plan, the objectives of the meeting, and the preferred format (in-person, teleconference, or written response).

3. Pre-IND meeting: If the meeting request is granted, we prepare for the meeting with the FDA. During the meeting, we will have the opportunity to discuss our candidate, the preclinical data, the proposed clinical trial design, and any concerns or questions we may have. Take note of the FDA’s feedback, as it can provide valuable guidance for the subsequent IND submission and overall drug development process.

4. Pre-IND written feedback: If FDA decides to provide written feedback to our pre-IND meeting request and following a review of the responses, we need further clarification based on their response, we can reach back to the PM in writing, stating we would appreciate raising a few clarifying questions as the responses provided differ from our pre-IND plan. The PM will request that we submit the questions in writing. In this follow-up request, we should ask for a 30-minute conference call and FDA will grant it if time allows. Otherwise, FDA will send a follow-up written response. Our questions should be very focused and specific. This call is not intended to be a pre-IND meeting. Take note of the FDA’s feedback, as it can provide valuable guidance for the subsequent IND submission and overall drug development process.

5. Address the FDA’s feedback: After the pre-IND meeting, carefully review the FDA’s comments and recommendations. Make any necessary adjustments to our therapy development plan, clinical trial design, and other aspects of the process based on their feedback.

6. Prepare the IND application: Use the information gathered during the pre-IND process to draft a comprehensive IND application, which should include detailed information on the drug candidate, preclinical study results, the proposed clinical trial design, chemistry, manufacturing, and controls (CMC) information, and any other required information. Make sure to address the FDA’s feedback from the pre-IND meeting in our IND application.

7. Submit the IND application: Once our IND application is complete, submit it to the FDA for review. If the FDA deems our application to be complete and satisfactory, they will grant us the clearance to proceed with clinical trials.

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