Pfizer saw positive topline results of its investigational gene therapy for adults with hemophilia A in a Phase III study (AFFINE). The therapy, giroctocogene fitelparvovec, reduced patients’ bleeds for at least 15 months and was generally well tolerated. It comprises a bio-engineered AAV6 capsid and a modified B-domain deleted human…

Boston-based hC Bioscience will target specific forms of hemophilia A with its lead candidate therapy, HCB-101, an anticodon engineered transfer (t)RNA. The company announced this week at the World Federation of Hemophilia 2024 World Congress in Madrid that they would be targeting the blood condition and that preclinical results for…

The first gene therapy for hemophilia A has finally reached the U.S. This week the FDA approved BioMarin’s Roctavian (valoctocogene roxaparvovec), an adeno-associated virus (AAV) vector-based gene therapy for the treatment of adults with severe hemophilia. The treatment reduced means annualized bleeding rate from 5.4 bleeds per year at baseline…

The potential transformation of hemophilia treatment through gene therapy was reaffirmed last week when results from the pivotal trial of CSL’s Hemgenix (etranacogene dezaparvovec-drlb) for hemophilia B appeared in the New England Journal of Medicine (NEJM). The same issue featured a positive report on two-year follow up of a trial…

Pfizer today announced very positive results from a Phase III study of fidanacogene elaparvovec, an investigational gene therapy, for the treatment of adult men with moderately severe to severe hemophilia B. This caps off a breakthrough year for hemophilia B treatment, as the first gene therapy for this blood clotting…

Belief BioMed’s BBM-H901, the first intravenously injectable hemophilia B gene therapy product developed and tested in Asia, showed very promising results in a Phase I study. Findings suggest that BBM-H901 is safe one year after infusion. Factor IX coagulation (FIX:C) concentration was sufficient to prevent bleeding events and minimize the…

Currently there are 51 companion diagnostic (CDx) tests approved by the U.S. Food and Drug Administration (FDA). The vast majority of these are used to recommend targeted therapies for hematological malignancies and solid tumors. In fact, of the 170 approved indications listed on the FDA website for companion diagnostics, only…

Kärt Tomberg, PhD, has a vision to express every recombinant protein at the levels required to implement its intended therapeutic effect. “There are proteins in the biotechnology field that need to be expressed in much greater abundance than they are now,” Tomberg told Inside Precision Medicine. “It might not sound…

Sickle cell disease (SCD) will be the first focus of the U.S.’s new Cell and Gene Therapy (CGT) Access Model, which aims to increase access to cell and gene therapies for vulnerable populations. Gene therapies are some of the most expensive treatments in the world. The Centers for Medicare &…

Precision medicines are gaining increasing traction in healthcare. Here are five companies driving the field with recent approvals in RNA therapeutics, cell and gene therapies, and more. Medicines have traditionally been given to patients in a one-size-fits-all approach, with many patients failing to benefit and experiencing side effects. This challenge…

By William A. Haseltine Regenerative medicine has emerged as a promising treatment option for chronic medical conditions. Specifically, cell and gene therapies, innovative forms of regenerative medicine, have shown great potential in personalizing and targeting disease treatment. These cutting-edge therapies leverage a patient’s cells and genes to repair and regenerate…

Depending on which research report is referenced, there are anywhere from a couple hundred to more than 600 bispecific antibodies (bsAbs) in preclinical and clinical development by pharma and biotech companies around the world. Nine of the 12 bsAbs that have received approval globally target cancer. But after the first…

After decades of research, cell and gene therapies are gaining traction in the biotech industry. Here are five investor-favored private startups with products already in clinical development. While conventional medications are often effective at controlling symptoms or modifying a disease, there are many conditions where their benefits are limited, such…

A little over a decade ago, a paper was published in Science1  that made science fiction a reality. Emmanuel Charpentier, Jennifer Doudna, and colleagues reported that they had identified a means of harnessing an element of a bacterial immune system to carry out genome editing in a way that was…

Everyone who knows her assumes that Nicole Paulk, PhD, has been working on improvements to the adeno-associated virus (AAV) platform for the past few years. They’re not wrong, but she’s also been working on a secret project that was unveiled at the American Society of Gene and Cell Therapy (ASGCT)…