ASC Therapeutics Partners with Vigene for Manufacturing Support for Hemophilia A Gene Therapy Program

Marta Figueiredo PhD avatar

by Marta Figueiredo PhD |

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ASC Therapeutics is partnering with Vigene Biosciences to support the manufacturing process of its current and future gene therapy clinical platforms — including its ongoing hemophilia A program.

“We are proud to welcome Vigene, a global leader in gene therapy process development and GMP [Good Manufacturing Practices] manufacturing with a proven track record and expertise in the field of viral vector manufacturing, to become an integral part of ASC long-term plan,” Ruhong Jiang, MD, ASC’s founder and CEO, said in a press release.

Hemophilia A is caused by mutations in the F8 or clotting factor VIII (FVIII) gene, which contains instructions to produce FVIII, an essential blood coagulation protein. People with hemophilia A are treated mainly with regular injections of a lab-made version of FVIII, administered into the bloodstream to prevent bleeding.

Gene therapy has the potential to target the underlying cause of this disease by delivering a functional copy of the F8 gene to patients.

ASC’s lead gene therapy candidate for hemophilia A, called ASC-618, uses a modified and harmless version of an adeno-associated virus (AAV) to deliver a working copy of the F8 gene to liver cells, where FVIII is mainly produced.

Thus, ASC-618 has the potential to promote a sustained production of FVIII — overcoming its deficit in hemophilia A and reducing both the need for prophylactic, or preventive, FVIII replacement therapy and the occurrence of bleeding events.

To improve ASC-618’s therapeutic effectiveness, ASC last year acquired exclusive rights to Expression Therapeutics’ liver-targeting F8 gene therapy technologies.

These technologies consist of specific genetic sequences that are added to the F8 gene to boost the production of FVIII in liver cells. They were shown to offer exceptional potency to this gene therapy in preclinical models of hemophilia A.

Jiang predicts that ASC’s genetic platform technology “is going to change the way serious diseases are treated in the future.” He said ASC-618 has shown “remarkable potency data” in hemophilia A preclinical studies.

“Our partnership provides ASC access to Vigene’s world-class team with expertise for both [DNA and virus] manufacturing as well as high-caliber [quality control and assurance] teams,” Jiang said.

“Vigene is excited to become a long-term [manufacturing] partner for ASC,” said Zairen Sun, MD, Vigene’s president and CEO. He said Vigene has already built multiple state-of-the-art facilities for its collaboration with ASC.

“We are well positioned to support ASC for both early-stage and commercial virus production,” Sun said. “This partnership will ensure that all ASC clinical deliverables are achieved in a timely fashion.”

ASC has already met with the U.S. Food and Drug Administration (FDA) to prepare for the filing of an investigational new drug application (IND) to test ASC-618 for hemophilia A.

According to ASC’s website, the company already has submitted an orphan drug designation application for ASC-618 for the same indication. The site also notes that clinical trials evaluating ASC-618 in hemophilia A patients are expected to begin this year.