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Gene Therapy

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...

Gene Therapy

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...

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A Nonviral Approach to Generate Transient Chimeric Antigen Receptor T Cells Using mRNA for Cancer Immunotherapy

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Published on: February 21, 2025

Velimogene aliplasmid.

Heloisa P Soares¹, Jose Lutzky

¹Department of Medicine, Mount Sinai Medical Center, 4300 Alton Road, Miami Beach, FL 33140, USA.

Expert Opinion on Biological Therapy

|April 7, 2010

Summary

This summary is machine-generated.

Velimogene aliplasmid shows promise as a cancer immunotherapy, demonstrating anti-tumor activity and a favorable safety profile in melanoma patients. Further investigation is ongoing for other cancer types.

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Area of Science:

Oncology
Immunotherapy
Gene Therapy

Background:

Cancer immunotherapy research has a long history with recent advancements driving renewed interest.
Intralesional plasmid gene transfection is an emerging strategy to enhance anti-tumor immune responses.

Purpose of the Study:

To review the role of velimogene aliplasmid (Allovectin-7) as an immunotherapeutic agent.
To discuss preclinical and clinical trial data for velimogene aliplasmid.

Main Methods:

Review of preclinical data.
Analysis of Phase I, II, and III clinical trials.
Evaluation of safety and efficacy in melanoma and other malignancies.

Main Results:

Velimogene aliplasmid induces anti-tumor responses in a subset of melanoma patients with locoregional and limited distant metastases.
Clinical trials indicate velimogene aliplasmid is safe with minimal adverse events.
Limited data suggests potential in other malignancies.

Conclusions:

Velimogene aliplasmid exhibits activity in melanoma with local and limited distant disease.
The agent possesses an excellent safety profile.
Ongoing studies are exploring its efficacy in additional cancer types.