On Oct. 13, the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee unanimously recommended Spark’s Luxturna in a vote of 16 to 0.
On Oct. 13, the U.S. Food and Drug Administration (FDA)’s Cellular, Tissue and Gene Therapies Advisory Committee unanimously recommended Spark Therapeutics’ Luxturna (voretigene neparvovec) in a vote of 16 to 0. Luxturna is a gene therapy to treat vision loss for a rare inherited retinal disease (IRD) caused by biallelic RPE65.
If approved, it would be the first full gene therapy approved by the FDA. The only other gene therapy yet approved by the FDA is for Novartis’ Kymriah for childhood leukemia.
The news gave a boost to at least three other biotech companies focusing on gene therapies.
1. Bluebird bio
Bluebird is involved in both immuno-oncology and gene therapy, more similar to Novartis’ Kymriah, where the T-cells are drawn from the patient, engineered, and infused back into the patient. The company claims extensive expertise in viral vector design and manufacturing and transduction. Its gene therapy pipeline is focused on severe genetic and rare diseases including cerebral adrenoleukodystrophy (CALD), transfusion-dependent beta-thalassemia, and severe sickle cell diseases.
Since its initial public offering (IPO) in 2013, Bluebird stock is up 438 percent.
2. Abeona Therapeutics
Headquartered in Dallas, Texas, Abeona focuses on developing therapies for rare genetic diseases. The company is developing next generation adeno-associated virus (AAV) gene therapies for central nervous system (CNS) disorders and lysosomal storage diseases. Initial programs are focused on Mucopolysaccharidosis (MPS) IIIA and IIIB, also known as Sanfilippo syndromes type A and type B.
The company announced yesterday that it is offering and selling shares of its common stock in an underwritten public offering. The net proceeds will be used for working capital and corporate purposes.
Benzinga notes that the company stock has been up 234 percent since the beginning of 2017.
3. Voyager Therapeutics
Headquartered in Cambridge, Mass., Voyager focuses on the development of therapies for severe neurological diseases like advanced Parkinson’s disease, monogenic forms of amyotrophic lateral sclerosis (ALS), Friedreich’s ataxia, Huntington’s disease, frontotemporal dementia and Alzheimer’s disease, and severe, chronic pain. It is utilizing adeno-associated virus (AAV) vector engineering for gene therapies.
The company announced today that it will present several studies at the Congress of the European Society of Gene and Cell Therapy (ESSGCT) this week in Berlin, Germany. Included is the presentations is one related to the company’s ongoing Phase 1b trial of VY-AADC01 in advanced Parkinson’s disease.
The company’s stock has been up 82 percent over the past year.
Although the Sparks nod of approval has bolstered gene therapy companies, the risk does not appear to have changed. It suggests that gene therapy may become a viable treatment methodology, but it’s still a field in its infancy and the companies involved in it will be volatile as a result. The Sparks committee vote was based on dramatic trial results, although there are still concerns as to whether the gene therapy is permanent or will require subsequent treatments. Meanwhile, Sparks stock is up 68.83 percent since the beginning of the year, and if the FDA ultimately approves its gene therapy, which is likely, will probably continue upward.
