Referred to as the Metropolis of Brotherly Love, Philadelphia, Pennsylvania, now has a brand new monicker—Cellicon Valley—to honor the cell and gene therapies that corporations equivalent to Spark Therapeutics are bringing to it.


Affectionally named “Cellicon Valley,” Philadelphia is the birthplace of cell and gene remedy and residential to greater than 30 corporations devoted to cell and gene remedy analysis. Philadelphia-based Spark Therapeutics grew to become the primary to deliver a U.S. Meals and Drug Administration (FDA)-approved gene remedy to market and has been increasing its footprint on this biotech hotspot for gene and cell remedy ever since, in response to Chief Scientific Officer Federico Mingozzi, Ph.D.

Headquartered in Philadelphia, Pennsylvania, Spark Therapeutics helps town turn out to be a premier spot for cell and gene remedy.

In 2013, Spark Therapeutics was based because of the technological and scientific know-how developed on the Youngsters’s Hospital of Philadelphia (CHOP). Now a part of the Roche Group, the corporate is dedicated to additional rising Philadelphia as a world hub of cell and gene remedy. With formidable progress plans, the corporate stays targeted on extending its scientific platform and distinctive competencies to guage and choose a portfolio of gene therapies concentrating on illnesses of the retina, hemophilia A, lysosomal storage problems, and neurodegenerative illnesses. They at the moment have 4 packages in medical trials advancing therapies to deal with hemophilia A, Pompe illness, and choroideremia.

Chief Scientific Officer Federico Mingozzi, Ph.D.

“With Roche’s worldwide attain and sources, we’re nearer than we’ve ever been earlier than to fulfilling our imaginative and prescient of dwelling in a world the place no life is proscribed by genetic illness,” Mingozzi says. Though many gene remedy research give attention to genetic illnesses, Mingozzi notes, there’s an urge for food to increase gene remedy to extra frequent indications.

Regardless of the challenges of 2020, there was super progress at Spark. The corporate now employs greater than 500 individuals and has a rising footprint in West Philadelphia. Spark has employed greater than 130 workers because the begin of 2020 and doesn’t intend to decelerate as the corporate heads into the upcoming yr. A part of that growth introduced Senior Analysis Affiliate Phillip Value, Ph.D. to Spark in February. Drawn by the corporate’s ardour and dedication to treating genetic illnesses, Value recollects pondering, “I knew I needed to be a part of this.”

On the time he was employed by Spark, Value had lately accomplished his doctorate in neuroscience at Emory College and was serving as a visiting researcher on the Mayo Clinic in Jacksonville, Florida. His robust conviction about the necessity to deliver extra therapies to extra sufferers dwelling with genetic illness, in tandem with a seamless interviewing course of, propelled Value to make the leap from Florida to Pennsylvania.

Senior Analysis Affiliate Phillip Value, Ph.D.

Regardless of working just about, Value says he was capable of get plugged into the corporate rapidly and discover “a powerful relationship between the employer and the workers, who inspired each private {and professional} progress.” Value mentions that the corporate additionally made vital efforts to grasp and reply to the altering dynamics ensuing from the pandemic, equivalent to working remotely. He has benefitted from the robust strains of communication from Spark’s management and its distinctive worker packages equivalent to half days each different Friday, which have enabled him to step away from work and chill out whereas studying extra about Philadelphia.

Breaking Boundaries

Though Value skilled some hesitancy in transitioning from academia to business, that disappeared when he discovered that all through the Spark workforce “persons are devoted to the training, listening, and innovation wanted to deliver the perfect therapies to sufferers.” Value appreciates that prime degree of motivation. “Gene remedy is not only about therapeutic targets of the longer term,” he notes. “That is right here now, and the door is open!” At Spark, he finds much more pleasure since being part of Roche offers “a platform to achieve globally.”

With expertise in finding out spinal muscular atrophy, an inherited dysfunction, Value matches proper in with Spark’s central nervous system (CNS) group, which has therapies in its pipeline for different genetic illnesses, together with Huntington’s illness and Batten illness. Already, he describes the surroundings as “very dynamic and primarily based on useful processes that assist domesticate concepts.” Plus, he rapidly felt like a part of the group, the place he says, “everybody has a voice.”

That is the hope: Philadelphia will proceed to draw and develop the group of biotechnology consultants and turn out to be one of the vital hubs for gene and cell remedy on the planet.

Federico Mingozzi, chief scientific officer, Spark Therapeutics

Science and town

The thriving life sciences business in Philadelphia additionally appealed to Value. Greater than 42,000 Philadelphians work in life sciences and town is now rated the fifth largest analysis and growth (R&D) hub within the nation, boasting $10.5 billion spent yearly in R&D, in response to statistics offered by Life Sciences Pennsylvania. Plus, worldwide data agency EqualOcean experiences, “Greater than 700 life science corporations have chosen to come back to Philadelphia to develop their merchandise.”

Along with CHOP, the area is residence to various different world-class life sciences establishments, together with the College of Pennsylvania Perelman College of Medication, Temple College, and the Drexel College Faculty of Medication. The mixture of Philadelphia’s tutorial establishments with the thriving surroundings it offers for biotech start-ups has resulted in a superb system for networking and collaborating, in response to Value.

Assay growth scientist Stephanie Kutza at work in Spark’s labs.

Mingozzi agrees. He added that Spark is one in every of a cluster of many lively corporations in gene and cell remedy which are making a vibrant biotech heart within the metropolis. “Having a hub attracts expertise from different cities and retains the expertise that’s already right here,” Mingozzi says. For instance, he notes that Philadelphia is a spot the place individuals come for coaching after which keep for work. Total, “it’s an unimaginable success story for Philadelphia, and the sector of gene and cell remedy is driving this success,” he says.

Nonetheless, Mingozzi sees this as only the start for Philadelphia. “You’ll see that the driving force of this hub can be a few corporations, like Spark, and that folks educated in Philadelphia will begin new corporations right here,” he says. “Then gene remedy will turn out to be a significant contributor for the native financial system.”

This numerous metropolis, nevertheless, presents far more than science. Value lives downtown, which he describes as “a really full of life and galvanizing environment.” As a historical past buff, he enjoys exploring Philadelphia with its many vital websites, together with Independence Corridor, the Liberty Bell and the Betsy Ross Home. Pondering of the structure, museums, and parks, Value has nothing however appreciation for the Metropolis of Brotherly Love, saying “it’s actually fairly lovely right here.”

However its magnificence, much more biotech progress lies forward in Philadelphia’s future. As Mingozzi sees it, “That is the hope: Philadelphia will proceed to draw and develop the group of biotechnology consultants and turn out to be one of the vital hubs for gene and cell remedy on the planet.”

Spark ranks among the many high employers in Science Careers’ 2020 Prime Employer survey. Read more

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