Gene therapy refers to any procedure that aims to treat or alleviate a disease by genetically modifying the cell of a patient. The material to be transferred may consist of genes, gene segments, or oligonucleotides and target cells may be normal cells, cancerous cells, immune mediated cells, or pluripotent stem cells.Unlike conventional treatments such as chemotherapy, gene therapy combines reduced treatment duration (one time treatment in some cases) with higher chances of cure in oncology. This novel therapeutic approach requires direct delivery of genetic material to the patient or in vitro genetic modification of patient cells and subsequent delivery to the patient, which is an uncharted territory in oncology and will necessitate the implementation of new regulatory guidelines and restructuring of the existing treatment algorithms in various oncology indications.
Currently only small size biotech and pharmaceutical companies dominate the pipeline for cancer gene therapies. With J&J having the exclusive worldwide rights to develop and commercialize Geron’s imetelstat and AstraZeneca the only large pharmaceutical company with an in-house gene therapy in late stage clinical development; Merck & Co. and BMS are evaluating their respective in-house CPI therapies with many of the Phase II and III candidates in the cancer gene therapy pipeline.
KOLs see a huge potential for cancer gene therapies, particularly in combination with immune CPIs. To this end, there is a significant opportunity for developing combination regimens as half of the Phase III pipeline is evaluated as a monotherapy and only one candidate is evaluated in combination with a CPI in their pivotal studies.
The report "Gene Therapy in Oncology", provides an overview of the current competitive landscape of gene therapies in multiple oncology indications, the regulatory framework concerning their clinical development and commercialization in the 8MM (US, France, Germany, Italy, Spain, UK, Japan, and China).
In addition, the report covers in detail the late stage clinical development of cancer gene therapies in the 8MM, providing key opinion leader (KOL) insight on the impact of cancer gene therapies in various treatment paradigms in the oncology space as well as payer perspective on the cost and reimbursement of novel cancer gene therapies.
- The author’s Gene Therapy in Oncology combines KOL and payer insight with in-house analyst expertise and research to provide an insight-rich look at cancer gene therapies in the 8MM.
- Overview of Cancer Gene Therapy: What are the gene transfer methods and strategies used in oncology.
- Cancer Gene Therapy in the 8MM: What is the regulatory framework concerning development and approval of cancer gene therapies in the 8MM and what are the currently marketed gene therapies in the oncology space in the 8MM.
- Clinical Development of Gene Therapies in Oncology: What are the late stage pipeline cancer gene therapies.
- Clinical Trial Mapping and Design: How are gene therapies positioned in the oncology space: what are the oncology indications they are targeting and which other therapeutic agents are they being evaluated in combination with.
- Market Outlook for Cancer Gene Therapies in Oncology: When are cancer gene therapies expected to be marketed for various oncology indications, what are some of the most promising candidates, and what are some challenges that cancer gene therapies are expected to face following their launch.
- Reimbursement of Gene Therapies in Oncology: What are the challenges associated with reimbursement of novel cancer therapies and some strategies to contain the cost of cancer gene therapies.
- Develop business strategies by understanding the trends shaping and driving the gene therapy in the oncology space in the 8MM.
- Formulate effective sales and marketing strategies by understanding the current competitive landscape and by analyzing the portfolios of various competitors.
- Identify areas of unmet need within the cancer gene therapy market to help drive research and development towards future market opportunities.
- Gain insights to help plan and design your clinical trials.
- Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments, and strategic partnerships.
- Identify key pricing and reimbursement strategies.
1.1 Table of Contents
1.3 Related Reports
1.4 Upcoming Related Reports
2. Executive Summary
2.1 Key Findings
2.2 KOL Insight on Cancer Gene Therapy Competitive Landscape
3. Overview - Cancer Gene Therapy
3.1 What is Gene Therapy?
3.2 Gene Transfer Methods and Vectors Used for Gene Therapy
3.3 Viral Vectors
3.4 Non-Viral Vectors
3.5 Gene Therapy Strategies Employed in Oncology
4. Cancer Gene Therapy in the 8MM
4.1 Global Regulatory Agencies’ Definitions of Gene Therapy
4.2 Cancer Gene Therapy in the US
4.3 Cancer Gene Therapy in the EU
4.4 Cancer Gene Therapy in Japan
4.5 Cancer Gene Therapy in China
4.6 Timeline of Clinical Development in Gene Therapy in the 8MM
4.7 Currently Marketed Gene Therapies in Oncology
5. Pipeline Assessment in the 8MM
5.1 Cancer Gene Therapy Pipeline in the 8MM
5.2 Pipeline Products - Phase III
5.3 Pipeline Products - Phase II
5.4 Mologen’s Lefitolimod
5.5 Idera’s IMO-2125
5.6 Geron’s Imetelstat
5.7 Oncotelic’s Trabedersen
5.8 VBL Therapeutics’ VB-111
5.9 Sillajen’s JX-594
5.10 Tocagen’s Toca 511
5.11 Advantagene’s ProstAtak
5.12 MarsalaBiotech’s E-10A
5.13 Advaxis’ AXAL
6. Clinical Trials Mapping and Design
6.1 Clinical Trial Mapping of Late Stage Gene Therapy Pipeline
6.2 Clinical Trial Mapping by Tumor Type
6.3 Clinical Trial Duration by Indication
6.4 Combination Partner by Therapy Area
6.5 Ongoing Clinical Trials of Cancer Gene Therapy Pipeline
7. Payer Perspective on Cancer Gene Therapies
7.1 Current Oncology Space
7.2 Challenges Associated with Reimbursement of Novel Cancer Therapies
7.3 Strategies to Tackle the Cost of Cancer Gene Therapies
7.4 A Novel Mechanism to Cover the Cost of Cancer Gene Therapies
8. Market Outlook
8.1 Phase III Cancer Gene Therapy Pipeline
8.2 Key Launch Dates for Phase III and II Cancer Gene Therapy Pipeline
9.2 Primary Research
9.3 About the Authors
- Advaxis Immunotherapies
- Ascend Biopharmaceuticals
- Bio-Path Holdings
- Bristol-Myers Squibb
- Cold Genesys
- CRISPR Therapeutics
- Daiichi Sankyo
- Editas Medicine
- Geron Corp
- Idera Pharmaceuticals
- Intellia Therapeutics
- Johnson & Johnson
- Marsala Biotech
- Merck & Co
- Mologen AG
- MTG Biotherapeutics
- Oncolytics Biotech
- Shanghai Sunway Biotech
- Shenzhen SiBiono GeneTech
- Sillajen Biotherapeutics
- SynerGene Therapeutics
- Takara Holdings
- Vascular Biogenics
- Ziopharm Oncology