CPHI NORTH AMERICA 2018

‘Cellicon’ Valley? Philadelphia has the capabilities to advance cell and gene therapies, says exec

By Flora Southey

- Last updated on GMT

GettyImages/7activestudio
GettyImages/7activestudio
Philadelphia, Pennsylvania has the logistics, capabilities, and expertise required to advance next-generation therapies in the US, says industry expert.

The term "Cellicon Valley" – a reference to California’s high-tech innovation hub – was coined by a Tmunity Therapeutics co-founder in response to Philadelphia’s stronghold on gene and cell therapy development in the US.

Philadelphia-headquartered Tmunity specialises in T-cell therapy development and CRISPR technology for anti-cancer treatments.

“If we’re lucky enough to bring in these next-generation products, then why can’t we rival another valley over in California?” ​CEO Usman Azam asked delegates during a panel discussion at CPhI North America yesterday.

CEO of the preclinical contract research organisation (CRO) Absorption Systems Patrick Dentinger agreed that Philadelphia has made its mark in the cell and gene space.

Philadelphia has a strong history in this therapy area, he said, “from the Philadelphia chromosome identification to the real first trial for a gene therapy that didn’t go so well here.”

The Philadelphia chromosome was discovered in 1959 at the University of Pennsylvania School campus. The unusually short chromosome is linked to a genetic abnormality in a chromosome of leukemia cancer cells.

Dentinger also made reference to a trial​ conducted in 1999 by the University of Pennsylvania. Eighteen-year-old Jesse Gelsinger – who suffered from a rare metabolic disease – died of a strong immune reaction to the experimental gene therapy treatment.

It is remarkable how the city has recovered from a situation that “would normally take out an industry and put everything on hold for a long time,” ​said Dentinger.

But the same city has come back to deliver breakthrough drugs, he added.

The US Food and Drug Administration (FDA) in December last year approved​ its first gene therapy developed by Pennsylvania-headquartered Spark Therapeutics.

Spark’s one-time gene therapy treatment Luxturna (coretigene neparvovec-rzyl) is indicated to treat patients with an inherited form of vision loss.

In February​, Adaptimmune Therapeutics announced it had made the first SPEAR-T cells for a patient at its facility in Navy Yard, Philadelphia. The firm’s SPEAR (specific peptide enhanced affinity receptor) platform is used to make T-cells that target and kill cancer.

For WuXi AppTec’s Alan Moore, Philadelphia has the expertise, capabilities, and logistics required to advance cell therapies.

The contract manufacturing organisation (CMO) has a facility dedicated to allogeneic and autologous cell-based therapeutics located just 15 minutes from the airport.

There is incredible logistics. The capabilities here and the tech here is a big driver," ​said Moore. “WuXi wouldn’t be investing hundreds of millions of dollars if this was the wrong location.” 

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