For most people, a small cut on the hand or foot isn’t a big deal. They can slap a Band-Aid on it and get on with life. But for those with hemophilia, even the smallest cut or bruise can lead to severe bleeding and other health issues. Caused by a mutation of genes that provide the ability to make clotting proteins, hemophilia occurs in about one of every 5,000 male births. Although rare, it can occur in females as well. And for many of these patients, hemophilia also causes progressive joint deterioration or repeated bleeding into their joints.
Current gene therapies for hemophilia – while exciting – are limited because they are only for patients who don’t experience immune response issues after receiving a therapeutic infusion. Plus, most therapies are designed to work for patients who have either hemophilia A or B without inhibitors, but none of the therapies treat both types of the disease with or without inhibitors.
But now, GeneVentiv Therapeutics is developing the first universal gene therapy for all types of hemophilia, including the most difficult cases. By working to bring breakthrough therapies to market, this Carolina-affiliated startup is also instilling expanded hope in hemophilia patients who wrestle with the daily realities of their life-threatening, inherited disorder.
“These patients suffer from tremendous problems, including neurological problems, gastrointestinal problems and joint deterioration, which reduces the quality and the duration of their lives,” says Damon Race, CEO of GeneVentiv. “Until now, there’s a group of patients that have had no hope of benefiting from exciting gene therapies that are in development. We’re able to give these patients new hope with a first-in-class gene therapy that can treat all types of hemophilia, delivering patients from these problems and rescuing them from a lifetime of weekly infusions or repeated injections. If we can give them a treatment and give them hope, we’ve got an opportunity to really change lives.”
A spinoff from the University of North Carolina at Chapel Hill’s Gene Therapy Center, GeneVentiv’s hemophilia treatment is based on research from Dr. Chengwen Li, whose novel technology potentially offers a treatment for all types of hemophilia. An associate professor of pediatrics, Li leads a research lab – based at the Gene Therapy Center – that is focused on rare disease gene therapy.
“By using Factor Va in the common pathway to restore homeostasis in hemophilia patients, we can offer the promise of a universal treatment for all patients by bypassing any missing upstream factors and inhibitors,” says Li.
Current treatment for hemophilia patients is a weekly infusion with the patient’s missing clotting factor, but roughly a third of patients will develop an inhibitor as a result of that treatment. Inhibitors are an immune system response to infused clotting factor concentrates, which renders standard replacement therapy ineffective. This presents real problems and complications, including neurological problems, gastrointestinal problems and joint deterioration. But GeneVentiv’s therapy promises to solve these challenges.
“Many gene therapies in development for either hemophilia A or hemophilia B can’t treat patients with neutralizing antibodies for their missing clotting factor,” says Race. “GeneVentiv’s gene therapy is the first gene therapy that bypasses any of the missing clotting factors and any inhibitors that patients have developed.”
For patients, the company’s life-changing treatment will potentially provide a long-awaited and lasting answer. That’s because GeneVentiv’s gene therapy is designed to be a single infusion. In theory, the protein will be expressed for the remainder of the patient’s life.
“We’re hoping our treatment will be curative. We’ve cured mice and have proof of concept studies demonstrating that we can cure hemophilia A and hemophilia B, with or without inhibitors,” says Race. “That’s how we can change lives. For these inhibitor patients that don’t have hope from any of the current, curative treatments that are in development, we can give them that hope.”
GeneVentiv is one of many Carolina-affiliated startups helping to raise the profile of Carolina at the national level for gene therapy. Race credits the state’s vibrant economy, educated workforce, universities and lower cost of living as part of the attractiveness for growing gene therapy startups in North Carolina.