Gene Therapy Pipeline Insights, 2023: Clinical Trials, Latest Approvals, Therapies, Key Companies | Candel Therapeutics, SillaJen Biotherapeutics, Wuhan Binhui Biotechnology, Virogin Biotech

Gene Therapy Pipeline Insights, 2023: Clinical Trials, Latest Approvals, Therapies, Key Companies | Candel Therapeutics, SillaJen Biotherapeutics, Wuhan Binhui Biotechnology, Virogin Biotech
Gene Therapy Pipeline Insights
“Gene Therapy Pipeline Insight, 2023” report by DelveInsight outlines a comprehensive assessment of the present clinical/non-clinical development activities and growth prospects across the Gene Therapy Market.

DelveInsight’s, “Gene therapy Competitive landscape, 2023,” report provides comprehensive insights about 250+ companies and 300+ drugs in Gene therapy Competitive landscape. It covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

The Gene Therapy Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers, acquisition, funding, designations, and other product-related details.

Recent Developments in Gene Therapy Market:

  • June 2022: Transgene and BioInvent International Collaboration with MSD In June 2022, Transgene and BioInvent International announced a clinical trial collaboration and supply agreement with MSD, the tradename of Merck & Co. This partnership aims to evaluate the gene therapy BT-001 in combination with MSD’s anti-PD-1 therapy KEYTRUDA (pembrolizumab) in a Phase I/IIa clinical trial for treating patients with solid tumors.

  • April 2022: ImmVira Partnership with Roche In April 2022, ImmVira announced a cooperation agreement with Roche to establish a clinical research partnership. This collaboration will conduct clinical studies in the U.S. on the combination therapy of ImmVira’s MVR-T3011 IT and Roche’s MEK inhibitor cobimetinib to evaluate the safety and efficacy of this combination strategy.

  • March 2022: Targovax and Agenus Collaboration In March 2022, Targovax announced a collaboration with Agenus to use their proprietary vaccine adjuvant QS-21 STIMULON as an immune-stimulatory component of the TG vaccines for future development and commercialization.

  • February 2022: Calidi Biotherapeutics and Edoc Acquisition Corp. Merger In February 2022, Calidi Biotherapeutics, Inc. and Edoc Acquisition Corp. announced a definitive merger agreement. Upon closing the transaction, expected in the second quarter of 2022, the combined company will be named Calidi Biotherapeutics, Inc. and led by Allan Camaisa, CEO and Chairman of the Board. Additionally, the combined company’s common stock intends to list on the Nasdaq Capital Market.

  • January 2022: Transgene and PersonGen BioTherapeutics Collaboration In January 2022, Transgene and PersonGen BioTherapeutics announced a strategic collaboration to evaluate the feasibility and efficacy of combining PersonGen’s TAA06 CAR-T cell injection with intravenous (IV) administration of an armed gene therapy from Transgene’s Invir.IO™ platform in solid tumors, including pancreatic cancer and brain glioma. The collaboration aims to demonstrate the synergistic mechanisms to enhance CAR-T cell therapy. Under the agreement, Transgene will develop multiple new oncolytic virus (OV) candidates using its patented gene therapy backbone VVcopTK-RR- and Invir.IO™ technology platform for IV administration in combination with PersonGen’s TAA06 CAR-T injection. PersonGen will evaluate the combination’s efficacy in eliminating solid tumors in preclinical models.

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Gene Therapy Overview

Gene therapy is a technique that uses genes to treat, prevent, or cure diseases and medical disorders. This approach often involves adding new copies of a gene to replace or supplement a defective or missing gene in a patient’s cells. Gene therapy has been used to treat both inherited genetic diseases (e.g., hemophilia and sickle cell disease) and acquired disorders (e.g., leukemia).

Gene Therapy Mechanism of Action

Gene therapy works by introducing genetic material—DNA or RNA—into cells to alter the production of proteins. This alteration can correct imbalances caused by the overproduction, underproduction, or incorrect production of essential proteins within cells. The therapeutic genetic material, such as a functional gene, is delivered into the cell using a vector. 

Vectors in Gene Therapy

A vector acts as a delivery system to transport the new genetic material into cells. Viruses are often used as vectors because they are naturally adept at entering cells. Scientists modify these viruses by removing their harmful genes, enabling them to safely deliver the therapeutic genetic material. This modified viral vector then inserts the new genetic material into the target cells. Before being used in humans, all viral vectors undergo extensive safety testing.

Gene Therapy Applications

Gene therapy has shown promise in treating a variety of conditions, including:

– Inherited Genetic Diseases: Such as hemophilia and sickle cell disease, where the therapy can replace or repair faulty genes.

– Acquired Disorders: Such as leukemia, where gene therapy can modify immune cells to better fight cancer.

Gene Therapy Safety and Development

The development of gene therapy involves rigorous testing to ensure safety and efficacy. This includes multiple stages of preclinical and clinical trials to evaluate the therapeutic potential and identify any risks. The continuous advancements in gene therapy research hold significant potential for providing long-lasting treatments and even cures for many genetic and acquired diseases.

Gene therapy represents a cutting-edge approach to medicine, offering hope for conditions that were once considered untreatable. As research progresses, the potential for gene therapy to transform medical treatment continues to expand.

Gene Therapy Pipeline Analysis

The report provides insights into: 

The report provides detailed insights into emerging therapies in the Gene Therapy Market and the aggregate therapies developed by major pharma companies.

  • It accesses the different Gene Therapy therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development.

  • It outlines the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.

  • The report evaluates the drugs that are under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type. 

  • It navigates the major collaborations (company-company collaborations and company-academia collaborations), licensing agreements, financing details, data presentation by the pharma giants, and regulatory approval in the Gene Therapy Market.

The report is built using data and information traced from the researcher’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.  

Analysis of Emerging Gene Therapy by Phases

The report covers the emerging products under different phases of clinical development like –

  • Late-stage products (Phase III)

  • Mid-stage products (Phase II)

  • Early-stage product (Phase I) 

  • Pre-clinical and Discovery stage candidates

  • Discontinued & Inactive candidates

Key Companies in the Gene Therapy Market Include:Candel Therapeutics, SillaJen Biotherapeutics, Wuhan Binhui Biotechnology, Virogin Biotech, Replimune, Istari Oncology, Immvira Pharma, Lokon Pharma, ORCA Therapeutics, Beijing SyngenTech, Tasly Pharmaceuticals, Turnstone Biologics, BioInvent Transgene, Elicera Therapeutics, Orgenesis, Virttu Biologics, Imugene, Astellas Pharma, Mustang Bio, Virogin Biotech, Memgen, Replimune, Immvira Pharma, PsiOxus Therapeutics, VCN Biosciences, Sorrento Therapeutics, Calidi Biotherapeutics, Replimune, TILT Biotherapeutics, DNAtrix, and many others.


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Table of Contents

1. Report Introduction

2. Executive Summary

3. Gene Therapy Current Treatment Patterns

4. Gene Therapy – DelveInsight’s Analytical Perspective

5. Therapeutic Assessment

6. Gene Therapy Late Stage Products (Phase-III)

7. Gene Therapy Mid-Stage Products (Phase-II)

8. Gene Therapy Early Stage Products (Phase-I)

9. Pre-clinical Products and Discovery Stage Products

10. Inactive Products

11. Dormant Products

12. Gene Therapy Discontinued Products

13. Gene Therapy Product Profiles

14. Key Companies in the Gene Therapy Market

15. Key Products in the Gene Therapy Therapeutics Segment

16. Dormant and Discontinued Products

17. Gene Therapy Unmet Needs

18. Gene Therapy Future Perspectives

19. Gene Therapy Analyst Review  

20. Appendix

21. Report Methodology

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