Companies partner to help speed development of gene, cell therapies

New treatments ultimately could benefit people with AADC deficiency

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

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GenScript ProBio and Comprehensive Cell Solutions (CCS) are partnering to accelerate the development of new cell and gene therapies, with the goal of making these treatments affordable and quickly accessible to patients.

Such therapies could ultimately benefit people living with rare disorders such as aromatic l-amino decarboxylase (AADC) deficiency.

“With this collaboration, we will streamline and improve the [cell and gene therapy] supply chain with continuity and consistency our clients can rely on,” Tracy Kinjerski, vice president and general manager of CCS, a part of New York Blood Center Enterprises, said in a company press release.

“This unique one-stop-shop approach accelerates the journey from development to clinic and commercial supply, ultimately improving the lives of patients,” Kinjerski added.

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Upstaza, approved in Europe, is first gene therapy for AADC deficiency

In the last few years, cell and gene therapies have garnered substantial interest for a wide range of diseases, including AADC deficiency, a genetic disorder characterized by impaired communication between nerve cells in the body.

Rather than simply treating symptoms, these advanced therapies work to directly target the faulty genetic or cellular mechanisms that cause a disease. In so doing, these treatments can be a way of slowing, stopping or reversing disease progression.

Gene therapies seek to add, edit, or remove DNA to treat diseases with a known genetic cause. A cell therapy, meanwhile, provides cells to the body that replace, remove, or modify the function of diseased or damaged ones.

AADC deficiency is caused by mutations in the DDC gene that’s needed to make the AADC enzyme. That enzyme is important for producing important nerve signaling chemicals; its deficiency leads to severe developmental delays and other neurological symptoms.

One successful gene therapy has been developed to date for AADC deficiency.

Approved in the European Union and in the U.K., PTC Therapeutics’ Upstaza (eladocagene exuparvovec) is a gene therapy that delivers a healthy version of DDC to the body. The therapy allows a patient’s cells to produce their own functional AADC enzyme. It’s packaged into a viral carrier, or vector, that helps the genetic material be taken up by the body’s cells when delivered directly into the brain.

Upstaza is the only therapy available in Europe to treat the underlying cause of AADC deficiency. It’s not approved in the U.S., but PTC plans to file an application with regulatory authorities in the coming months.

No other gene or cell therapies are approved for AADC deficiency in any part of the world.

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Gene, cell therapies target underlying causes of disease

The new collaboration between GenScript ProBio and CCS will initially focus on helping drug developers — pharmaceuticals, biotechs, hospitals and government organizations — to advance cell and gene therapies for a range of indications, including blood cancers, immune disorders, and rare diseases.

This support will span the entire development process, from therapeutic development to clinical manufacturing to regulatory strategy and commercial manufacturing. A wide range of therapeutic technologies will be supported, according to the companies.

Our mutual dedication to quality, safety, and efficiency will expedite the development and commercialization of novel cell therapy treatments for patients.

By helping drug developers navigate any hurdles — such as issues with cost-effectiveness, quality assurance, regulatory processes, and large-scale production — that can slow the advancement of these types of therapies, the two companies hope to “accelerate and simplify the development and delivery of groundbreaking treatments to patients,” the release states.

The relationship relies on CCS’ expertise in cell therapy development and manufacturing, as well as on GenScript’s experience with development and manufacturing of antibody and protein based therapeutics, viral vectors, vaccines, and other therapeutic components.

“Joining forces … allows us to harness the combined experience and capabilities of both entities to propel innovation and uphold excellence within the cell therapy sector,” said Patrick Liu, PhD, chairman of the board of directors at GenScript.

“Our mutual dedication to quality, safety, and efficiency will expedite the development and commercialization of novel cell therapy treatments for patients,” Liu added.