Gene Therapy for Spinal Muscular Atrophy UPDATE

One of the Programs we have funded and are excited about is the SMA Gene Therapy program using the AAV9  Vector (Intravascular Delivery). The Sophia’s Cure Foundation has great faith in the very promising work being done at Nationwide Children’s Hospital and Ohio State University. The Team consisting of Lead Researcher Dr.Kaspar , Dr. Burgess, Dr. Kissel as well as Dr Mendell is second to none. The experience of Dr. Jerry Mendell working with the FDA  in advancing programs from Proof of Concept to clinical trials is very encouraging. We believe in this team and have helped to fund the creation of the actual Gene to be delivered to SMA Patients. Yesterday’s Pre Pre IND meeting with the Food and Drug Administration has offered us further encouragement in moving this program towards a formal application for human clinical trials. We must point out though, the Pre Pre Ind conversation is an informal communication with the FDA and is non-binding. Nevertheless this conversation was insightful into the path forward towards turning the AAV9 program into a viable therapy for the SMA patient population.

“We had our first interaction with the Food and Drug Administration regarding our gene delivery program for Spinal Muscular Atrophy on Thursday, February 24th, 2011 in what is termed a pre-pre IND (Investigational New Drug Application).  For this call, we presented pertinent information to the FDA regarding the status of our studies to date, which included pre-clinical efficacy and preliminary safety data.  We were impressed with the thorough, thoughtful and expert review we received from the agency in which the FDA was positive regarding our program, providing guidance for moving our  translational program forward.  It is important to note that these discussions were informal and non-binding, but certainly provided positive direction for us to move towards human studies.  Based on our conference call and review, we are planning to perform some further dosing and safety studies that will help guide the studies that will be required for our formal application.  This was an important step in our program and we are excited to continue to advance the gene delivery program forward to the clinic.” ~ Dr. Brian Kaspar Ph.D.


  1. So exciting! We have been praying for good news and it looks like things are going in the right direction. THank you Gaynor family for putting your all into raising money for research!

  2. Morgan Saunders says:

    GREAT NEWS! Alot of prayer is going up for Dr. Kaspar and his team. Keep up the good work :) We are so close!

  3. This is so great! Keep up the great work Dr.Kasper and your team. Praying every day.

  4. Oscar hernandez says:

    Couldn’t expect less! You see? Good guys always win, (so their families)… This proves a valid, reliable and competent bonding with hope. Let’s spread the word to the People in the South! Thanks!

  5. Ritu and Ritesh says:

    I give my heartiest congratulations to Sophia’s Cure Foundation for their gr8 efforts towards the cause.
    And pray to the ALL MIGHTY for positive results of the hardwork and dedication of Dr.Kaspar , Dr. Burgess, Dr. Kissel and Dr Mendell and their team

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