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Adeno-Associated Virus Vector Serotype 8 Containing The Human F9 Gene

This article explores the ongoing clinical trials of REGV131-LNP1265, an innovative gene therapy designed to treat Hemophilia B. This groundbreaking treatment uses an adeno-associated virus vector serotype 8 containing the human F9 gene to potentially improve clotting factor production in patients with this rare bleeding disorder. The trials aim to evaluate the safety, effectiveness, and long-term benefits of this novel approach in adult patients with Hemophilia B.

Table of Contents

What is REGV131?

REGV131 is a new experimental treatment for hemophilia B, a rare genetic bleeding disorder. It’s also known as ADENO-ASSOCIATED VIRUS VECTOR SEROTYPE 8 CONTAINING THE HUMAN F9 GENE.[1] This medication is designed to help patients with hemophilia B produce their own clotting factor, potentially reducing or eliminating the need for regular injections of clotting factor.

How Does REGV131 Work?

REGV131 is a type of gene therapy. It uses a harmless virus (adeno-associated virus serotype 8, or AAV8) to deliver a healthy copy of the F9 gene to the patient’s liver cells. The F9 gene is responsible for producing Factor IX, a crucial protein for blood clotting that is missing or defective in people with hemophilia B.[1]

This treatment is described as an “in vivo CRISPR-associated protein 9 (Cas9)-based gene insertion therapy.” This means that the gene editing occurs inside the patient’s body (“in vivo”) using a technology called CRISPR-Cas9, which can precisely insert the healthy F9 gene into the patient’s DNA.[1]

Who Can Receive REGV131?

The current clinical trial for REGV131 is designed for adult males with severe or moderately severe hemophilia B. To be eligible, patients must meet several criteria, including:[1]

  • Be 18 years of age or older
  • Have a confirmed diagnosis of severe or moderately severe hemophilia B
  • Be currently taking Factor IX prophylaxis (preventive treatment)
  • Have participated in a lead-in study period for at least 6 months

Some conditions may exclude a person from participating in the trial, such as:

  • History of Factor IX inhibitors
  • Pre-existing antibodies to the AAV8 virus
  • Significant liver disease
  • History of blood clots

Clinical Trial Details

The clinical trial for REGV131 is divided into two parts:[1]

  1. Part 1: This phase focuses on evaluating the safety and tolerability of REGV131, as well as determining the recommended dose for further development.
  2. Part 2A: This phase aims to assess the effectiveness of REGV131 at the recommended dose, particularly in terms of Factor IX activity and reduction in bleeding events.

The treatment is administered as a single intravenous (IV) infusion. Patients will be monitored for up to 104 weeks (2 years) after receiving the treatment.[1]

Potential Benefits

If successful, REGV131 could offer several benefits to patients with hemophilia B:[1]

  • Increased Factor IX activity in the blood
  • Reduced frequency of bleeding events
  • Decreased need for Factor IX replacement therapy
  • Potential for long-lasting or permanent improvement in clotting ability

Safety Considerations

As with any experimental treatment, there are potential risks and safety considerations:[1]

  • The clinical trial will closely monitor for any adverse events related to the treatment.
  • Patients will be checked for the development of antibodies against various components of the treatment.
  • The presence of the viral vector will be monitored in different bodily fluids over time.

It’s important to note that this is an early-stage clinical trial, and the full safety profile and effectiveness of REGV131 are still being evaluated.

Aspect Details
Study Type Phase 1/2, open-label
Intervention REGV131-LNP1265 (AAV8 vector containing human F9 gene)
Administration Single intravenous infusion
Primary Objectives Safety, tolerability, FIX activity, bleeding rate reduction
Key Inclusion Criteria Adult males with severe/moderately severe Hemophilia B, on FIX prophylaxis
Key Exclusion Criteria History of FIX inhibitors, pre-existing AAV8 antibodies, significant liver disease
Follow-up Duration Up to 104 weeks
Primary Endpoints Adverse events, FIX activity, annualized bleeding rate

FAQ

  • What is REGV131-LNP1265? REGV131-LNP1265 is a gene therapy that uses an adeno-associated virus vector serotype 8 (AAV8) containing the human F9 gene. It's designed to help the body produce clotting factor IX in patients with Hemophilia B.
  • Who is eligible for this clinical trial? The trial is open to adult males (18 years and older) with severe or moderately severe Hemophilia B, who have been on FIX prophylaxis and have participated in a lead-in study for at least 6 months.
  • How is the treatment administered? REGV131-LNP1265 is administered as a single intravenous (IV) infusion.
  • What are the main objectives of the trial? The trial aims to evaluate the safety and tolerability of REGV131-LNP1265, assess its effect on clotting Factor IX activity, and measure the reduction in bleeding events in patients with Hemophilia B.
  • How long will participants be monitored in the study? Participants will be monitored for up to 104 weeks (2 years) to assess long-term safety and effectiveness of the treatment.

Ongoing Clinical Trials on Adeno-Associated Virus Vector Serotype 8 Containing The Human F9 Gene

  • Study on the Safety and Effectiveness of REGV131 and LNP1265 for Adults with Hemophilia B

    Recruiting

    2 1 1
    Investigated diseases:
    Investigated drugs:
    France Germany Italy Spain

Glossary

  • Hemophilia B: A rare genetic bleeding disorder caused by a deficiency in clotting Factor IX, which can lead to prolonged bleeding and spontaneous internal bleeding.
  • Factor IX (FIX): A protein in the blood that helps in the clotting process. People with Hemophilia B have low levels or absence of this protein.
  • Gene therapy: A technique that uses genes to treat or prevent disease. In this case, it involves introducing a functional F9 gene to help the body produce clotting Factor IX.
  • Adeno-associated virus (AAV): A small virus that can infect humans but is not known to cause disease. It's often used as a vector in gene therapy to deliver genetic material into cells.
  • Vector: In gene therapy, a vector is a vehicle used to deliver genetic material into cells. In this trial, AAV8 is used as the vector.
  • Prophylaxis: Preventive treatment. In Hemophilia, it refers to regular infusions of clotting factor to prevent bleeding episodes.
  • Annualized Bleeding Rate (ABR): A measure used to assess the frequency of bleeding events in patients with hemophilia over a year.
  • Chromogenic substrate assay: A laboratory test used to measure the activity of clotting factors in the blood.
  • CRISPR/Cas9: A gene-editing technology used in this therapy to insert the functional F9 gene into the patient's DNA.

References

  1. http://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-regv131-and-lnp1265-for-adults-with-hemophilia-b/