Sangamo Therapeutics to Take Back Rights to Hemophilia A Therapy

Sangamo Therapeutics Regains Gene Therapy Program Rights

Sangamo Therapeutics, Inc. (NASDAQ: SGMO), a leading genomic medicine company, has recently announced a significant development in its ongoing quest to treat hemophilia A. The company has received news that it will regain the full rights to giroctocogene fitelparvovec, an investigational gene therapy designed for adults suffering from moderately severe to severe hemophilia A. This change follows Pfizer Inc.'s decision to end their collaboration on this promising therapy, a move that has left many anticipating the next steps for both companies involved in this critical area of research.

An Important Shift in Collaboration

This transition signifies a critical juncture for Sangamo, who co-developed giroctocogene fitelparvovec in partnership with Pfizer. The announcement came after Pfizer decided to halt its collaboration, which had been aimed at helping develop and commercialize this innovative therapy. Sangamo is now focusing on its options to move the program forward autonomously, including the possibility of establishing new partnerships. The decision by Pfizer to terminate the agreement reflects its choice not to proceed with the Biologics License Application (BLA) and Marketing Authorization Application (MAA) submissions, which are key steps toward commercializing the therapy.

Positive Trial Results Preceding Termination

In mid-2024, Pfizer had reported encouraging topline results from the Phase 3 AFFINE trial, which evaluated the efficacy and safety of giroctocogene fitelparvovec. These results indicated that the therapy met both primary and secondary endpoints successfully, establishing its potential as a groundbreaking treatment. However, just months later, despite the positive data presented at prominent conferences, Pfizer decided to withdraw, leading to uncertainty regarding the future of the therapy.

Understanding the Impact of This Decision

“Giroctocogene fitelparvovec has shown great promise as a revolutionary treatment for hemophilia A patients,” stated Sandy Macrae, CEO of Sangamo Therapeutics. He expressed disappointment over Pfizer's decision, especially given the favorable pivotal clinical trial outcomes. Nevertheless, Sangamo is unwavering in its commitment to pursue the most effective path forward for this vital therapy.

Plans to Move Forward

Moving ahead, Sangamo is determined to explore all options to continue developing giroctocogene fitelparvovec. The company aims to find the right partner who understands the complex landscape of genomic medicine and can assist in bringing this innovative treatment to patients in need. In addition to this, Sangamo will continue advancing its wholly owned neurology genomic medicine pipeline, including the development of its Fabry gene therapy program, which it hopes to submit for BLA consideration by the end of 2025.

Details Surrounding the Program Transition

The formal termination of the agreement with Pfizer is set for April 21, 2025. During this transition, all clinical trial participants will still receive monitoring and support as needed. Sangamo remains optimistic about its existing partnerships with Genentech and Astellas, which could bolster new opportunities for moving forward with its developmental pipeline.

About Hemophilia A

Hemophilia A is a rare genetic disorder that leads to prolonged bleeding due to a deficiency in clotting Factor VIII. This condition affects approximately 25 out of every 100,000 male births globally, with a significant percentage of cases classified as moderate to severe. Patients living with hemophilia A face challenges that require continuous management and treatment, underscoring the importance of innovative therapies like giroctocogene fitelparvovec.

About Sangamo Therapeutics

Sangamo Therapeutics is at the forefront of genomic medicine, striving to translate groundbreaking scientific advancements into effective treatments for those afflicted by serious neurological diseases. With a firm belief in the potential of its therapies to address severe medical needs, Sangamo's efforts extend beyond hemophilia A to various programs designed to change the lives of patients worldwide. The company continues to forge partnerships, seek investments, and utilize cutting-edge technology to advance its mission.

Frequently Asked Questions

What is giroctocogene fitelparvovec?

Giroctocogene fitelparvovec is an investigational gene therapy product candidate designed to treat adults with moderately severe to severe hemophilia A.

Why did Pfizer terminate the collaboration with Sangamo?

Pfizer decided to end the collaboration due to its choice not to move forward with the Biologics License Application and Marketing Authorization Application submissions for the treatment.

What are Sangamo’s plans moving forward?

Sangamo aims to explore new collaborations to advance giroctocogene fitelparvovec and continue its ongoing research into other gene therapies.

What impact does hemophilia A have on patients?

Hemophilia A can lead to prolonged bleeding, requiring ongoing management and treatment, which significantly affects patients' quality of life.

How is Sangamo positioned in genomic medicine?

Sangamo Therapeutics is dedicated to translating groundbreaking science into transformative treatments for diseases, with a strong focus on cooperation and innovation in the genomics space.

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