Pfizer's "mini" gene therapy begins treatment of the first patient

Pfizer's "mini" gene therapy begins treatment of the first patient

April 16, 2018 Source: WuXi PharmaTech

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Recently, Pfizer announced that it has initiated Phase 1b clinical trials of patients with Duchenne muscular dystrophy (DMD) treated with "mini" dystrophin gene therapy PF-06939926. The first patient was on March 22 The day received treatment. Early data for the study is expected to be completed in the first half of 2019.

DMD is a serious hereditary disease characterized by progressive muscle degeneration and weakness. The disease mainly affects boys and usually begins to manifest symptoms during the early 3-5 years of childhood. Muscle weakness first affects the muscles of the buttocks, pelvis, thighs, and shoulders, and then affects the muscles of the arms, legs, and torso. By adolescence, patients often lose walking ability, and the heart and respiratory muscles are also affected, eventually leading to premature death. DMD is the most common form of muscular dystrophy worldwide, with one in every 3,500 to 5,000 male infants.

DMD is caused by the absence of dystrophin, a protein that helps keep muscle cells intact. In the absence of this protein, muscle cells will deteriorate. Pfizer's PF-06939926 is a recombinant mini-dystrophin that is under the control of a human muscle-specific promoter. The AAV9 capsid was selected as the delivery system for its potential to target muscle tissue.

This time, a multicenter, open-label, non-random, incremental dose study will recruit approximately 12 boys aged 5-12 years with DMD to assess the safety of a single intravenous drip of PF-06939926. Tolerance, dystrophin expression and distribution, and muscle strength, quality, and function. As part of the screening process, potential therapeutic candidates will also be tested to confirm that antibodies to the AAV9 capsid and T cell responses to dystrophin are negative.

“On behalf of DMD patients and family members, I would like to thank Pfizer for the major steps to promote a potential transition treatment for boys with this terrible disease,” said Ms. Debra Miller, CEO and founder of CureDuchenne. “We are in gene therapy. The momentum seen in the field underscores the mature opportunities that drive scientific development. These boys now have very limited treatment options. Through collaboration and ongoing dialogue with companies like Pfizer, we hope to find ways to change the treatment of DMD patients. ”

"The investment in this trial represents a culmination of years of research by Pfizer and academic medical centers, and has been supported by a group of DMD patients to advance a project that could change the path of this debilitating disease. Dr. Greg LaRosa, senior vice president and chief scientist of Pfizer's Rare Diseases Research Department, said: "We listened to the patients' opinions and we know that they urgently need treatment options. With this in mind, we have designed a potential for scientific advancement. The mini-dystrophin gene is delivered to in vivo treatments to address the underlying cause of DMD, regardless of the mutation. This trial will assess the safety of this gene therapy and provide valuable data to prove that it slows or blocks The potential impact of DMD progress."

We expect Pfizer's groundbreaking trial to bring effective treatment to patients with DMD.

Reference materials:

[1] PFIZER DOSES FIRST PATIENT USING INVESTIGATIONAL MINI-DYSTROPHIN GENE THERAPY FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY

[2] In First Trial of New Gene Therapy Program, Pfizer Doses First Duchenne Muscular Dystrophy Patient

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