ASGCT President's Message—January 2018

2017 continued to be a landmark year for gene therapy with the announcement on December 19, that the FDA approved Luxturna (voretigene neparvovec-rzyl) for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. Luxturna developed by Spark Therapeutics uses an adeno-associated virus (AAV) vector to deliver the normal human RPE65 gene to retinal cells. This was a particular milestone as Luxturna is not only the first directly administered in vivo gene therapy approved in the United States but also the first approval to target a genetic disease and the first to use AAV.

This will likely be the first of many approvals using AAV as there are a number of other AAV products currently in clinical testing in eye gene therapy and in other diseases such as hemophilia. Dr. Kathy High who will deliver the George Stamatoyannopoulos lecture at the upcoming Annual Meeting will provide an overview of the development and future directions of AAV gene therapy strategies to treat genetic diseases.

Read more:

Full statement on the approval of Luxturna

The History of Luxturna

In the spirit of New Year’s resolutions, ASGCT is developing new initiatives for 2018. The Board of Directors recently passed a budget that will allow for additional funds to be allocated for patient education and supporting our trainee members. In 2017, ASGCT collaborated with the Hemophilia Federation of America for a series of two webinars. This year, the Society will continue this effort with a renewed investment in resources for educating the patient and patient advocate community about gene therapies that are approved or in clinical trials.

Additionally, ASGCT plans to fulfill the commitments made in the 2016 strategic plan with regard to supporting and investing in the next generation of gene and cell therapy researchers with input from the Trainee Council. Expanding on current trainee support, the Society will bring the number of Excellence in Research Awards from six at $1,000 each to 15. The Society will add funding and for up to seven fellowships or trainee exchange grants and seven career development grants valued at $25,000 which will correspond to the primary abstract categories to ensure coverage of all areas in our field.

I urge you all to make plans to attend the Annual Meeting in Chicago, IL, May 16-19, 2018. Visit the Annual Meeting website to register and book your stay at the Hilton Chicago. Additionally, the deadline for abstract submission is January 31, at 5pm Central. You will not want to miss out on the opportunity to share your research at the Annual Meeting. Associate Members who are first and presenting authors of an abstract receive free registration.

Please also consider submitting a nomination for an ASGCT Award. Nominations for the Outstanding Achievement Award, Outstanding New Investigator Awards, and the Sonia Skarlatos Public Service Award must be submitted by tomorrow, January 19.

The President's Message is a part of ASGCT's bi-monthly newsletter, The Vector. You can find the rest of The Vector on the ASGCT site or have it sent directly to your inbox by becoming a member of ASGCT.

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