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Lentiviral Vector Containing The Human Abca4 Gene

This article discusses the clinical trials of a promising drug called SAR422459, which contains a lentiviral vector with the human ABCA4 gene. This innovative therapy is being studied for its potential in treating Stargardt’s macular degeneration, a rare genetic eye disorder. The trials aim to evaluate the long-term safety, tolerability, and biological activity of this treatment in patients with Stargardt’s disease.

Table of Contents

What is LENTIVIRAL VECTOR CONTAINING THE HUMAN ABCA4 GENE?

LENTIVIRAL VECTOR CONTAINING THE HUMAN ABCA4 GENE, also known by its product name SAR422459, is an innovative medical treatment currently being studied for its potential to help patients with a specific eye condition[1]. This treatment is classified as an advanced therapy, specifically a gene therapy product[1].

In simple terms, this treatment uses a special virus (called a lentivirus) as a delivery system to introduce a healthy copy of the ABCA4 gene into the cells of the eye. The ABCA4 gene is important for proper eye function, and when it’s faulty, it can lead to vision problems[1].

What Medical Condition Does It Treat?

The primary medical condition that this treatment targets is Stargardt’s macular degeneration, also known as Stargardt’s disease[1]. This is a genetic eye disorder that affects the retina, the light-sensitive tissue at the back of the eye. Stargardt’s disease typically causes progressive vision loss, often beginning in childhood or young adulthood[1].

Stargardt’s disease falls under the broader category of congenital, hereditary, and neonatal diseases, specifically affecting the eye[1].

How Does It Work?

The treatment works by using a lentiviral vector, which is a type of virus that has been modified to safely deliver genetic material into cells. In this case, the vector carries a healthy copy of the ABCA4 gene[1].

The treatment is administered as a suspension for injection, likely directly into the affected area of the eye (subretinal injection)[1]. Once inside the eye cells, the healthy ABCA4 gene can potentially help correct the genetic defect that causes Stargardt’s disease, potentially slowing down or stopping the progression of vision loss[1].

Current Clinical Trial Information

A clinical trial is currently underway to study the long-term effects of this treatment. The trial is titled “An Open Label Study to Determine the Long Term Safety, Tolerability and Biological Activity of SAR422459 in Patients with Stargardt’s Macular Degeneration”[1].

The main objectives of this trial are:

  1. To evaluate the long-term safety and tolerability of SAR422459 in patients with Stargardt’s macular degeneration[1].
  2. To assess the biological activity of the treatment, which means to see if it’s having the desired effect in the body[1].

The trial is focusing on several key aspects:

  • Safety: Researchers are closely monitoring the incidence of adverse events (side effects) in patients who receive the treatment[1].
  • Ocular health: They’re looking for any clinically important changes in various measures of eye health[1].
  • Disease progression: The trial is assessing whether the treatment can delay the degeneration (breakdown) of the retina[1].

Who Is Eligible for the Trial?

The trial has specific criteria for who can participate. Eligible participants must:

  • Have been previously enrolled in a specific earlier study (protocol TDU13583)[1].
  • Have received a subretinal injection of SAR422459 as part of that earlier study[1].
  • Have completed the earlier study to Week 48 or undergone an early discontinuation visit[1].
  • Provide informed consent, which means they must agree to participate after being fully informed about the study[1].

People who did not receive SAR422459 as part of the earlier study are not eligible to participate in this follow-up trial[1].

Safety and Monitoring

Safety is a primary concern in this trial. The researchers are carefully monitoring participants for any side effects or adverse reactions to the treatment. This includes:

  • Recording any adverse events that occur[1].
  • Conducting regular ocular (eye) safety assessments to check for any changes in eye health[1].
  • Monitoring the progression of the disease to see if the treatment is helping to slow or stop vision loss[1].

Future Research and Data Sharing

The company conducting this research, Sanofi, has committed to sharing the data from this study with other qualified researchers. This means that after the study is complete, other scientists may be able to access the data to conduct further analyses or to help inform future research[1].

However, it’s important to note that any shared data will be anonymized to protect the privacy of the study participants. This means that any information that could identify individual participants will be removed before the data is shared[1].

Aspect Details
Drug Name SAR422459
Active Substance Lentiviral vector containing the human ABCA4 gene
Condition Treated Stargardt’s macular degeneration
Study Type Open-label, long-term safety and tolerability study
Administration Method Subretinal injection
Primary Endpoint Incidence of Adverse Events
Secondary Endpoints Changes in ocular safety assessments, delay in retinal degeneration
Orphan Drug Status Yes (EU/3/09/720)

FAQ

  • What is SAR422459 and how does it work? SAR422459 is a suspension for injection containing a lentiviral vector with the human ABCA4 gene. It's designed to deliver a functional copy of the ABCA4 gene to cells in the retina, potentially helping to slow or stop the progression of Stargardt's disease.
  • What is the main objective of the clinical trial? The main objective of the trial is to evaluate the long-term safety and tolerability of SAR422459 in patients with Stargardt's macular degeneration.
  • Who is eligible to participate in this clinical trial? Eligible participants must have been enrolled in a previous study (protocol TDU13583), received a subretinal injection of SAR422459, and completed that study to Week 48 or undergone an early discontinuation visit.
  • What are the primary endpoints of the study? The primary endpoint of the study is the incidence of Adverse Events, which helps researchers assess the safety of the treatment.
  • Is SAR422459 considered an orphan drug? Yes, SAR422459 has been granted orphan drug designation by the European Union (designation number EU/3/09/720), which means it's intended to treat a rare disease or condition.

Ongoing Clinical Trials on Lentiviral Vector Containing The Human Abca4 Gene

  • Study on the Long-Term Safety of SAR422459 for Patients with Stargardt’s Disease

    Not recruiting

    2 1 1
    Investigated diseases:
    Investigated drugs:
    France

Glossary

  • Lentiviral Vector: A type of virus-based tool used to deliver genetic material into cells. In this case, it's used to carry the ABCA4 gene.
  • ABCA4 Gene: A gene that provides instructions for making a protein involved in the visual cycle. Mutations in this gene can cause Stargardt's disease.
  • Stargardt's Macular Degeneration: A genetic eye disorder that causes progressive damage to the macula, leading to vision loss.
  • Subretinal Injection: A method of delivering medication directly under the retina of the eye.
  • Orphan Drug: A medication developed specifically to treat a rare medical condition.
  • Adverse Events: Any unfavorable and unintended sign, symptom, or disease temporarily associated with the use of a medical treatment or procedure.
  • Biological Activity: The effect of a drug on living tissue or organisms.
  • Phase I/II Follow-up Study: A study that continues to monitor participants from earlier phase trials to gather more data on long-term effects and safety.

References

  1. http://clinicaltrials.eu/trial/study-on-the-long-term-safety-of-sar422459-for-patients-with-stargardts-disease/