Spark Therapeutics Inc(NASDAQ: ONCE) recently reported that the U.S. Food and Drug Administration’s (FDA) Cellular, Tissue and Gene Therapies Advisory Committee has approved LUXTURNA™ (voretigene neparvovec), for treating patients with vision loss on the back of the confirmed biallelic RPE65-mediated inherited retinal disease (IRD).

This approval of LUXTURNA would lead to the investigational adeno-associated viral (AAV) vector gene therapy to patients with vision loss on the back of confirmed biallelic RPE65-mediated IRD. LUXTURNA clinical program comprises patient data which shows efficacy for up to four years on endpoints including bilateral multi-luminance mobility test (MLMT) score change and full-field light sensitivity threshold (FST) testing, with observation ongoing.

On the other side, Jefferies analysts, Michael Yee gave a Buy rating and maintained their $95 price target. As per the analyst viewpoint, the group would proceed with just three to five centers when its therapy is launched and then expand to eight to 10 centers. As per the analyst interview with Management, they see several LCA patients that are eligible for immediate treatment.

Moreover, insurance payers are also willing to pay a lump sum payment for the group’s Luxturna as it is an ultra-rare disease. The Jefferies analysts also notes the “early and positive discussions” with payers on the economic value of its Hemophilia therapies.

The group expects their new hemophilia data between Dec. 9 and 12 for SPK-8011 for Factor VIII. They are targeting for at least five to seven patients depending on dosing schedule. Their Hem B program is also in the process of being transferred to Pfizer who would start the pivotal phase 3 program and run a global study to support global registrations.

The group made a licensing agreement with Genethon, which is a non-profit research and development organization of gene therapies for orphan genetic diseases from research to clinical validation, for the development and commercialization of an adeno-associated viral (AAV) gene therapy targeting the liver to address a rare genetic disease. The group also appointed Federico Mingozzi, Ph.D., as chief scientific officer.