Experimental gene-silencing therapy offers hope for hepatitis B cure
Chronic hepatitis B is a long-lasting condition caused by the hepatitis B virus (HBV) that affects about 240 million people worldwide. Most people infected show no symptoms for years, but when symptoms do appear, they can include fatigue, jaundice and abdominal pain. Current antiviral treatments are designed to keep the virus under control, but they are not a cure, and medications are usually…
Chronic hepatitis B, affecting around 240 million people globally, is a condition caused by the hepatitis B virus (HBV) that often shows no symptoms for years. Existing antiviral treatments help control the virus but do not offer a cure, and patients usually need to take medication for life. This has driven scientists to explore new ways to eliminate the infection without lifelong medication.
One promising experimental therapy being studied is CRMA-1001, developed by nChroma Bio. This therapy uses a genetic package to target and silence viral DNA in human liver cells and mouse models.
CRMA-1001 consists of a delivery vehicle carrying a guide RNA to locate viral DNA and instructions for a gene-silencing protein, all encased in protective lipid bubbles. When introduced to hepatitis B-infected liver cells, the therapy effectively reduced two viral proteins by 98%. Moving from liver cells to mice carrying HBV DNA, researchers administered a single dose of CRMA-1001, which significantly lowered one viral marker in the blood by over 1,000 times.
This reduction persisted for six months, and administering three monthly doses further suppressed the virus. Six months after the final dose, up to 90% of the mice had undetectable levels of hepatitis B surface antigen in their blood, and their viral DNA levels were no longer measurable.
To determine if CRMA-1001 could reach the liver in a human-like body, the scientists tested it in healthy monkeys. The therapy primarily targeted the liver and caused only a temporary increase in liver enzymes at the highest repeated dose. Subsequent tests in uninfected human liver cells from healthy donors revealed no unintended changes in gene expression or DNA methylation elsewhere in the human genome.
While these findings are encouraging, the researchers stress that animal models may not fully replicate how hepatitis B behaves in humans.
The study authors believe their results support initiating clinical trials of CRMA-1001 in humans.
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