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Rare Disease Start-Up: Spark Therapeutics’ Gene Therapy Programs

November 3, 2014

11/04/14: Corrected Post Title From Fate Therapeutics to Spark Therapeutics

This Blog Post reviews the new start-up, Spark Therapeutics and the company’s gene therapy programs.

In October 2013, Children’s Hospital of Philadelphia (CHOP) invests $50 million in Spark Therapeutics, a new biotech start-up that develops gene therapies for orphan diseases. Since the company’s launch a year ago, $83 million in financings have been secured from eight life sciences institutional investors.

Spark Therapeutics assumes control of two gene therapy clinical trials that started at CHOP:

•   Phase III study for Inherited Retinal Dystrophies (IRDs) caused by autosomal recessive mutations in the RPE65 gene – most advanced product candidate, SPK-RPE65

•   Phase I/II study for Hemophilia B – product candidate SPK-FIX.

Spark Therapeutics’ initial product programs use Adeno-Associated Virus (AAV) vector technology, which is designed to “encapsulate a genetic sequence that has been demonstrated to be an effective vehicle for delivering genetic material into targeted cells.” Data for the Phase III clinical trial for IRDs is expected to be reported in the second half of 2015, with the submission of a FDA Biologics Licensing Application (BLA) in 2016. The initiation of human trials for Hemophilia B with product candidate SPK-FIX is expected in the first half of 2015. Spark Therapeutics has worldwide rights to both gene therapy products.

Spark Therapeutics plans on providing treatment for more genetic diseases in the future – other rare forms of blindness, blood disorders, and neurodegenerative diseases. Several gene therapies have received FDA orphan designation, but none has yet been approved. In Europe, a gene therapy called Glybera receives approval for the treatment of Lipoprotein Lipase Deficiency in 2012.

In September 2014, FierceBiotech, names ST as one of 2014’s “Fierce 15 biotechnology companies”.

References

Spark Therapeutics Pipeline

Wall Street Journal 09/14/14 article “Gene Therapies Push Investors to Examine New Areas of Human Body“.

Please Note: “Gene Therapy” National Institute of Health [Public domain] | Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

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