SPARK Therapeutics History Foretells Its Triumph in Gene Therapy


Spark (NASDAQ: ONCE) is on its way to get an FDA approval of its first gene therapy for a rare genetic condition that causes blindness in children. The firm’s gene therapy product,SPK-RPE65 for inherited retinal dystrophies is designed to be given only once to produce the expected cure.

In recent news, Spark is financing through a secondary public offering. The firm plans to use the proceeds for:

Planning, preparing and “infrastructure-building” related to the potential launch of SPK-RPE65;

-Further development of its experimental hemophilia A therapy;

-Continued development of large-scale manufacturing processes and expansion of its   manufacturing capabilities;

Non-clinical and clinical development of Spark’s other pipeline candidates, and;

For working capital and other general corporate purposes including potential in-licensing deals and acquisitions.


Spark has granted the underwriters of the offering a 30-day option to purchase up to an additional 525,000 shares at the public offering price

J.P. Morgan Securities, Cowen and Co., and RBC Capital Markets are acting as book-running managers for the offering. Stifel, Nicolaus & Co. Inc. and SunTrust Robinson Humphrey Inc. are acting as lead managers for the offering.

That was the most recent news coming from SPARK.

We wrote an article about Spark, which was posted under Today Highlights at the Prohost Website:  on January 12, 2016

Prohost Observations

We liked the story behind Spark’s creation. Yet, its history explains the reasons for its success and for our enthusiasm.

Here is the story. 

Spark Therapeutics spun out of Children Hospital of Philadelphia (CHOP) and was launched with a $50 million capital commitment from the West Philadelphia pediatric medical center. The creation of Spark followed years of commitment by CHOP and the company’s founding team to move gene therapy forward during a time when most other gene therapy firms were halting their gene therapy programs.

Indeed, gene therapy research had slowed In the early 2000s, and venture-capital firms gave up on financing what appeared to be at the a failing task by the firms that struggled to produce breakthrough gene therapy therapeutics.

In 2004, CHOP established the Center for Cellular and Molecular Therapeutics as a place for conducting gene therapy translational research and manufacturing.  S a matter of fact, the project has provided solutions to many of the problems associated with gene therapy.

Spark was made to control the two clinical trials CHOP’s was conducting. One is a late-stage study of a gene therapy for inherited blindness caused by gene mutations, and the other is an early-stage study as a potential hemophilia B therapy.

The two products tested became Spark’s pipeline, which also added gene therapy programs to address neurodegenerative diseases and other hematologic disorders and forms of inherited blindness. Many of the Center for Cellular and Molecular Therapeutics’ leaders became managers, or served as scientific advisors to Spark.

Now we are waiting for the approval of SPK-RPE65, the one shot treatment expected to prevent blindness in patients who carry the mutation that causes the inherited retinal dystrophy.

It is a great step forwards that might be followed by other treatments for other inherited diseases.

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