The American Society of Gene and Cell Therapy (ASGCT) has unveiled a new online resource designed to keep patients, caregivers, and the public apprised of the latest in gene and cell therapies, including those intended for spinal muscular dystrophy (SMA).
Gene Therapy 101 is the organization’s debut release from its new Patient Education Program, designed by ASGTC committee volunteers in tandem with patient advocacy groups. The program’s aim is to provide information on therapies’ status and potential.
Through February, the organization will also explore five diseases, specifically chosen because of the promising statuses of their gene and cell therapies, according to a press release. The diseases and release dates are: SMA, Feb. 1; X-linked myotubular myopathy, Feb. 12; leukodystrophy, Feb. 15; blood disorders, Feb. 22; and inherited retinal disorders, Feb. 28.
This is particularly relevant for SMA, because Zolgensma — developed by Novartis-owned Avexis — is on its way to potentially becoming the first gene therapy approved for the disease, with a U.S. Food and Drug Administration decision expected in May.
Now available online, Gene Therapy 101 discusses gene therapy basics; different approaches to gene therapy and editing, and cell therapy; and how gene therapies go from concepts to treatments. With the help of videos and downloadable graphics, it also provides a host of resources and frequently asked questions.
The site begins by laying out the basics — what gene therapy is, its history, and how it works, explaining what genes are and how gene therapy can help. It also defines what a rare disease is, lists disorders that have gene therapies, and describes the use of viral vectors.
A section on different approaches discusses how the biomedical research fields of gene therapy, cell therapy, and gene editing have a shared goal of treating diseases by altering the genetic makeup of the human body. Supported by a glossary of terms, it also describes how each approach changes genetic material in slightly different ways.
This section also discusses gene and cell therapy versus traditional medications as well as the challenges and benefits of the therapy, including funding and regulatory issues, optimal means of genetic material delivery, long-term effects and maintenance, and therapeutic agent design.
A third section explaining how therapies progress to treatments also discusses the role of the FDA, and the meaning and impact of federal breakthrough, fast track, regenerative medicine advanced therapy, and priority review designations. In addition, it delves into patient access and finding clinical trials.
The upcoming segment on disease-specific treatments opens with SMA, a disease caused by a change, or mutation, in a person’s DNA. In SMA patients, there’s a mutation in the SMN1 gene, which leads to a lack of SMN protein production, and ultimately to the loss of motor neurons. In humans, a second gene — SMN2 — can produce a small amount of SMN protein, but not enough to maintain muscle function.
Investigators have been working on ways to raise the levels of SMN protein in SMA patients. One approach is gene therapy, which scientists believe may be a way to treat the disease. This involves gene alteration, either by replacing a defective gene or adding a new one.
In addition to Avexis, two other companies are investigating gene therapy approaches for SMA, namely Genzyme (a Sanofi company) and Genethon Inserm.
The ASGTC is the nation’s chief professional membership organization for scientists, physicians, patient advocates, and other professionals with an interest in gene and cell therapy.
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