INDUSTRY UPDATE, cont.

of vascular injury to overcome the body's inability to form healthy clots.

The Phase 1/2 first-in-human open-label study in patients with Glanzmann was designed to evaluate HMB-001 for safety, tolerability, biomarkers such as FVII levels and bleeding time before and after HMB-001, and efficacy based on assessment of changes in bleeding frequency. Facilitated in collaboration with the UK-based clinical research organization Richmond Pharmacology, the trial is expected to expand into the US and other EU countries with initial data are expected in the second half of 2023.

Hemab is a clinical-stage biotech company developing next generation therapeutics for serious, underserved bleeding and thrombosis disorders. It is based in Denmark and the US and backed by Novo Holdings, RA Capital, and HealthCap.

Gene Therapy Registry

The World Federation of Hemophilia has developed a registry to monitor the long-term safety and efficacy of gene therapy for people with hemophilia: the WFH Gene Therapy Registry (GTR The launch of the WFH GTR coincides with the approval of gene therapy for hemophilia by both the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA).

This worldwide endeavor aims to collect important data on all patients who receive gene therapy, whether through a clinical trial or through a post-marketed product. Clinical data from around the world will be combined in this registry and used to answer critical questions on the long-term safety and efficacy of gene therapy in hemophilia.

The WFH GTR was developed in collaboration with the International Society on Thrombosis and Haemostasis (ISTH), the European Haemophilia Consortium (EHC), the US National Hemophilia Foundation (NHF), the American Thrombosis and Hemostasis Network (ATHN), PatientReported Outcomes Burdens and Experiences (PROBE) study group, manufacturers, and experts in the field of gene therapy. The WFH also partners with the United BioSource Corporation and Lifelink Systems for the technological development of the WFH GTR. The Gene Therapy

Registry is supported by Biomarin, CSL Behring, Pfizer, Spark (Founding visionary partners) and Takeda (Collaborating partner).

Gene editing

Scientists gathered at the Francis Crick Institute in London, for the Third International Summit on Human Genome Editing earlier this month. Five years after the last summit, CRISPR technology has continued to mature. The advent of CRISPR gene-editing technology, short for “Clustered Regularly Interspaced Short Palindromic Repeats”, has raised a number of ethical considerations, including the delivery of equitable access to genome editing therapies, ongoing research to optimize delivery systems for genome editing apparatus and delivery of measures to foster discussions regarding regulation, governance, public and patient engagement.

Many new advances in genome editing techniques were presented.

American chemist and biologist David Liu reported on findings to use “prime editing” to treat genetic conditions such as Huntington’s disease and Friedreich’s ataxia. Unlike CRISPR, which makes a double stranded cut in the DNA, prime editing induces a single stranded cut. This makes it more versatile and precise for targeted deletion and insertion of genetic sequences.

A somatic genome editing treatment for sickle cell disease is set to obtain regulatory approval in the US later this year. “Somatic” genome editing (which makes changes that are not heritable) is different to germline and heritable genome editing (which makes heritable changes). There were also reports of research using CRISPR technology to treat diseases including Duchenne muscular dystrophy, cancer, HIV/AIDS, heart and muscle disease and inborn errors of immunity.

In our area, Regeneron, with headquarters in Tarrytown and a facility in North Greenbush, is working on gene editing for hemophilia.