# Gene Therapies * **Definition:** Innovative treatments that involve altering, modifying, or manipulating the genes inside a patient's cells to treat or prevent diseases, particularly genetic disorders and cancers. * **Taxonomy:** Healthcare Topics / Gene Therapies ## News * Selected news on the topic of **Gene Therapies**, for healthcare technology leaders * 10.5K news items are in the system for this topic * Posts have been filtered for tech and healthcare-related keywords | Date | Title | Source | | --- | --- | --- | | 5/22/2025 | [**Navigating the Complexities of the Cell and Gene Therapy Landscape: Insights From the 2025 Advanced Therapies Report**](https://www.pharmacytimes.com/view/navigating-the-complexities-of-the-cell-and-gene-therapy-landscape-insights-from-the-2025-advanced-therapies-report) | [[Pharmacy Times]] | | 5/6/2025 | [**Vinay Prasad tapped to run FDA center that regulates vaccines, gene therapies**](https://www.statnews.com/2025/05/06/vinay-prasad-fda-center-for-biologics-evaluation-and-research-peter-marks-hhs-rfk-jr-makary/) | [[STAT]] | | 4/19/2025 | [**Advancing World-Class Healthcare Through Technology and Global Partnerships**](https://www.latimes.com/specialsupplements/one-world-media/story/taiwan-healthcare-technology-global-partnerships) | [[LA Times]] | | 2/21/2025 | [**Cell and Gene Therapies in Rare Disorders Market to Skyrocket Across the 7MM During the Forecast Period (2025-2034) - DelveInsight**](https://www.globenewswire.com/news-release/2025/02/21/3030613/0/en/Cell-and-Gene-Therapies-in-Rare-Disorders-Market-to-Skyrocket-Across-the-7MM-During-the-Forecast-Period-2025-2034-DelveInsight.html) | [[Globe Newswire]] | | 2/21/2025 | [**Cell and Gene Therapies in Rare Disorders Market to Skyrocket Across the 7MM ... - Yahoo Finance**](https://finance.yahoo.com/news/cell-gene-therapies-rare-disorders-180000095.html) | [[Yahoo Finance]] | | 2/18/2025 | [**Andelyn Biosciences Expands AAV Curator™ Platform Offering to Include Stanton Lab CNS Capsids through a License Agreement from the Broad Institute of MIT and Harvard**](https://www.prnewswire.com/news-releases/andelyn-biosciences-expands-aav-curator-platform-offering-to-include-stanton-lab-cns-capsids-through-a-license-agreement-from-the-broad-institute-of-mit-and-harvard-302379140.html) | [[PR Newswire]] | | 1/31/2025 | [**Biotech Market to Grow by USD 805.6 Billion (2025-2029), Boosted by Vendor Innovation ...**](https://www.prnewswire.com/news-releases/biotech-market-to-grow-by-usd-805-6-billion-2025-2029-boosted-by-vendor-innovation-with-ai-driving-market-transformation---technavio-302364467.html) | [[PR Newswire]] | | 1/30/2025 | [**Building a Playbook for Cell and Gene Therapies for Autoimmune Disease - PRWeb**](https://www.prweb.com/releases/building-a-playbook-for-cell-and-gene-therapies-for-autoimmune-disease-navigating-a-rapidly-evolving-environment-upcoming-webinar-hosted-by-xtalks-302363652.html) | [[PRWeb]] | | 1/29/2025 | [**Global Pharmaceutical R&D Outsourcing Market Research 2025 - Now Available - ResearchAndMarkets.com**](http://www.businesswire.com/news/home/20250129751453/en/Global-Pharmaceutical-RD-Outsourcing-Market-Research-2025-Now-Available---ResearchAndMarkets.com/?feedref=JjAwJuNHiystnCoBq_hl-RLXHJgazfQJNuOVHefdHP-D8R-QU5o2AvY8bhI9uvWSD8DYIYv4TIC1g1u0AKcacnnViVjtb72bOP4-4nHK5ieT3WxPE8m_kWI77F87CseT) | [[Business Wire]] | | 1/9/2025 | [**Global Biotechnology Market Outlook 2025: Driving Growth at 13.0AGR - PR Newswire**](https://www.prnewswire.com/news-releases/global-biotechnology-market-outlook-2025-driving-growth-at-13-0-cagr--marketsandmarkets-302346776.html) | [[PR Newswire]] | | 1/9/2025 | [**Global Biotechnology Market Outlook 2025: Driving Growth at 13.0AGR**](https://finance.yahoo.com/news/global-biotechnology-market-outlook-2025-153000473.html) | [[Yahoo Finance]] | | 12/29/2024 | [**Ocular Is Now Thoroughly Derisked (NASDAQ:OCUL) - The Total Pharma Tracker**](https://seekingalpha.com/mp/1202-the-total-pharma-tracker/articles/6102781-ocular-is-now-thoroughly-derisked) | [[Seeking Alpha]] | | 12/19/2024 | [**ICYMI: Highlights From AMCP Annual 2024**](https://www.ajmc.com/view/icymi-highlights-from-amcp-annual-2024) | [[AJMC]] | | 12/11/2024 | [**ASHP Midyear: High Costs of Gene Therapies Present Major Challenges to Implementation**](https://www.pharmacytimes.com/view/ashp-midyear-high-costs-of-gene-therapies-present-major-challenges-to-implementation) | [[Pharmacy Times]] | | 12/10/2024 | [**Multi-Million Drug Therapy Innovations Bring Challenges to Healthcare - Yahoo Finance**](https://finance.yahoo.com/news/multi-million-drug-therapy-innovations-145600846.html) | [[Yahoo Finance]] | | 12/4/2024 | [**Vertex, bluebird join new federal program to pay for gene therapies**](https://www.bizjournals.com/boston/news/2024/12/04/vertex-bluebird-cms-gene-therapy-cost-payments.html) | [[The Business Journals]] | | 11/11/2024 | [**Population Health Management Market to Grow by USD 22.55 Billion from 2024-2028 ...**](https://www.prnewswire.com/news-releases/population-health-management-market-to-grow-by-usd-22-55-billion-from-2024-2028--driven-by-rising-adoption-of-healthcare-it-report-with-ai-impact-on-trends---technavio-302300769.html) | [[PR Newswire]] | | 11/8/2024 | [**Cell and Gene Therapy CRO Market Research Trends and Global Forecasts, 2035 - Over 3,500 Trials are Evaluating Cell and Gene Therapies to Treat Myriad of Disease Indications - ResearchAndMarkets.com**](http://www.businesswire.com/news/home/20241108091390/en/Cell-and-Gene-Therapy-CRO-Market-Research-Trends-and-Global-Forecasts-2035---Over-3500-Trials-are-Evaluating-Cell-and-Gene-Therapies-to-Treat-Myriad-of-Disease-Indications---ResearchAndMarkets.com/?feedref=JjAwJuNHiystnCoBq_hl-RLXHJgazfQJNuOVHefdHP-D8R-QU5o2AvY8bhI9uvWSD8DYIYv4TIC1g1u0AKcacnnViVjtb72bOP4-4nHK5ieT3WxPE8m_kWI77F87CseT) | [[Business Wire]] | | 11/7/2024 | [**Gene Therapy Market to Grow by USD 6.74 Billion from 2024-2028, as Rise in Special Drug ...**](https://www.prnewswire.com/news-releases/gene-therapy-market-to-grow-by-usd-6-74-billion-from-2024-2028--as-rise-in-special-drug-designations-drives-demand-with-ai-powered-market-evolution---technavio-302297720.html) | [[PR Newswire]] | | 11/4/2024 | [**Cell and Gene Therapy CRO Market Research Trends and Global Forecasts, 2024-2035**](https://finance.yahoo.com/news/cell-gene-therapy-cro-market-104000689.html) | [[Yahoo Finance]] | | 9/30/2024 | [**Taysha Gene Therapies, Inc. (TSHA)**](https://finance.yahoo.com/quote/TSHA/news/) | [[Yahoo Finance]] | | 9/6/2024 | [**Taysha Gene Therapies Announces Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)**](https://finance.yahoo.com/news/taysha-gene-therapies-announces-inducement-120000927.html) | [[Yahoo Finance]] | | 8/28/2024 | [**Researchers call for individualized gene therapies to be more widely accessible**](https://markets.businessinsider.com/news/stocks/researchers-call-for-individualized-gene-therapies-to-be-more-widely-accessible-1033732323) | [[Business Insider Markets]] | | 8/13/2024 | [**5 Key Providers Taking Precision Medicine into the Cloud**](https://www.insideprecisionmedicine.com/topics/precision-medicine/5-key-providers-taking-precision-medicine-into-the-cloud/) | [[Inside Precision Medicine]] | | 6/24/2024 | [**Global Genomics Industry Research 2024: Rising Adoption of Personalized Medicines and Gene Therapies - Forecasts to 2031 - ResearchAndMarkets.com**](http://www.businesswire.com/news/home/20240624828195/en/Global-Genomics-Industry-Research-2024-Rising-Adoption-of-Personalized-Medicines-and-Gene-Therapies---Forecasts-to-2031---ResearchAndMarkets.com/?feedref=JjAwJuNHiystnCoBq_hl-RLXHJgazfQJNuOVHefdHP-D8R-QU5o2AvY8bhI9uvWSD8DYIYv4TIC1g1u0AKcacnnViVjtb72bOP4-4nHK5ieT3WxPE8m_kWI77F87CseT) | [[Business Wire]] | ## Topic Overview (Some LLM-derived content — please confirm with above primary sources) ### Key Players - **GenScript Biotech**: A company committed to advancing cell and gene therapy, hosting forums to foster collaboration and innovation in the field. - **Taysha Gene Therapies, Inc.**: A clinical-stage biotechnology company focused on developing AAV-based gene therapies for severe monogenic diseases. - **CRISPR Therapeutics**: A biotechnology company focused on gene editing technologies for various genetic disorders. - **AskBio**: A fully integrated gene therapy company with a diverse portfolio of clinical programs and proprietary capsids and promoters. - **GEMMABio**: A biotech company formed from the University of Pennsylvania's Gene Therapy Program, focusing on enhancing access to rare disease gene therapies. - **Dyno Therapeutics**: A biotechnology firm utilizing AI-driven platforms to develop next-generation AAV vectors for gene therapies. - **Senti Biosciences**: A company developing next-generation cell and gene therapies using its proprietary Gene Circuit platform. - **Key Player**: Medicare, Definition: A federal health insurance program that is expected to require pharmacist involvement in managing gene therapies. - **REGENXBIO**: A biotechnology company focused on developing gene therapies for rare genetic diseases. - **Vertex Pharmaceuticals**: A biopharmaceutical company that has developed gene therapies for sickle cell disease. - **Orna Therapeutics**: A biotechnology firm utilizing lipid nanoparticle delivery technology to enhance gene editing therapies, focusing on genetic diseases beyond the liver. - **Coave Therapeutics**: A biotechnology company specializing in gene therapies for central nervous system disorders. - **Fuse Vectors**: A biotechnology start-up focused on improving the manufacturing process of gene therapies. - **NewBiologix**: A company specializing in advanced technologies for the production of viral vectors for gene therapy, focusing on improving yields and reducing costs. - **Pharmaceutical Strategies Group (PSG)**: A healthcare technology firm that launched GeneCQ, a solution for healthcare payers to manage gene therapy exposure. - **Ascend Gene & Cell Therapies**: Specializes in scalable manufacturing for biotech projects, raising over £122 million to enhance quality and adaptability. - **Trogenix**: A company developing precision viral immunotherapy for aggressive cancers, particularly glioblastoma. ### Partnerships and Collaborations - **Taysha Gene Therapies and The University of Texas Southwestern Medical Center**: A strategic partnership aimed at advancing gene therapies for CNS diseases. - **Vertex Pharmaceuticals and bluebird bio**: Participating in the Cell and Gene Therapy Access Model to improve access to therapies for Medicaid patients. - **Belief BioMed and AskBio**: Strategic collaboration to develop gene therapies targeting diseases with high unmet medical needs using a liver-targeted approach. - **Integrated DNA Technologies and the Innovative Genomics Institute**: Collaboration to create CRISPR treatments for genetic disorders. - **ForCell**: A company focused on scaling new mechanical delivery technology for gene therapies, enhancing treatment accessibility. - **Andelyn Biosciences and Broad Institute**: Expanded agreement to include CNS capsids for gene therapy research and development. - **Dyno Therapeutics and Roche**: Partnership to develop AAV gene therapy vectors for neurological diseases, leveraging AI for enhanced vector properties. - **Novartis and Kate Therapeutics**: Acquisition to enhance Novartis' gene therapy offerings for inherited neuromuscular diseases. - **Astellas Pharma and Sangamo Therapeutics**: Entered a licensing agreement for the development of gene therapies targeting neurological diseases. - **Vertex Pharmaceuticals and Orna Therapeutics**: Collaborating to develop gene therapies for sickle cell disease and beta thalassemia, with Vertex providing $65 million upfront and potential milestone payments. - **Affinia Therapeutics and Forge Biologics**: Collaboration to provide cGMP manufacturing services for gene therapy AFTX-201 targeting BAG3 dilated cardiomyopathy. - **MIT Sloan School of Management, Boston Children's Hospital, Columbia University, N=1 Collaborative**: Collaborating to make gene and cell therapies more affordable and accessible globally. - **Bluebird Bio and Vertex Pharmaceuticals**: Agreements with CMS to participate in the Cell and Gene Therapy Access Model for sickle cell disease. - **Ginkgo Bioworks and Virica Biotech**: A strategic partnership to enhance AAV gene therapy manufacturing, aiming to reduce production costs and improve accessibility. - **Andelyn Biosciences and the Broad Institute**: License Agreement to enhance AAV CuratorTM Platform with MyoAAV plasmids for improved targeting of muscle tissue. - **GEMMABio and Oswaldo Cruz Foundation**: Collaboration to replicate manufacturing capabilities in Brazil for gene therapies targeting rare diseases. - **Teijin Limited and Hilleman Laboratories**: A strategic partnership to expand CDMO services in cell and gene therapy, targeting the Asia-Pacific market. - **Roche and Dyno Therapeutics**: A collaboration to develop novel AAV vectors for neurological diseases, leveraging Dyno's AI-driven platform. - **Emily Whitehead Foundation**: A non-profit organization advocating for access to advanced therapies, including CAR-T cell therapy. - **Pharmacists and Healthcare Systems**: The integration of pharmacists into healthcare systems is advocated to improve efficiency and patient care, particularly in managing complex gene therapies. ### Innovations, Trends, and Initiatives - **Advancements in Gene Therapies**: Significant progress in gene therapies for rare diseases, including FDA approvals for therapies targeting conditions like sickle cell disease and spinal muscular atrophy. - **Emerging Technologies**: Breakthroughs in genome editing technologies such as CRISPR-Cas9 and TALENs are enhancing the precision and efficiency of gene therapies. - **Gene and Cell Therapy Institute**: Launched by Mass General Brigham to translate scientific discoveries into clinical applications and FDA-approved treatments. - **Liver-Targeted Gene Therapies**: Emerging focus on liver-targeted approaches for gene therapies, as highlighted by the collaboration between Belief BioMed and AskBio. - **Nanoparticle Technology**: Developed by Johns Hopkins researchers for targeted gene treatment delivery, reducing patient burden and side effects. - **Gene Therapy Trials**: In 2023, 114 gene therapy trials were initiated in the US, with 77% sponsored by the healthcare industry. - **Synthetic Super-Enhancer Technology**: Trogenix's innovative approach to selectively target cancer cells while preserving healthy cells, aiming for curative therapies. - **Cell and Gene Therapy Access Model**: Introduced by CMS to improve access to gene therapies for underserved populations, particularly for sickle cell disease. - **Mechanical Delivery Methods**: Innovative technology developed to enhance gene therapies by using mechanical forces for gene modification without damaging cells. - **Patient-Centric Approaches**: Increasing focus on patient-centricity in the development and implementation of gene therapies. - **AI-Driven Gene Delivery Solutions**: Dyno Therapeutics' LEAP technology aims to optimize AAV capsids for improved therapeutic outcomes. - **GenScript Biotech Global Forum**: An event focusing on breakthroughs and future trends in cell and gene therapies, emphasizing the importance of patient advocacy and commercialization challenges. - **AI in Gene Therapy**: The use of AI to enhance the development of gene therapy vectors and improve delivery mechanisms, as seen in Dyno Therapeutics' partnership with Roche. - **GeneCQ**: A technology solution by PSG to help healthcare payers anticipate exposure to newly-approved gene therapies. - **Sickle Cell Disease Treatments**: Recent approvals of gene therapies for sickle cell disease, with ongoing discussions about outcomes-based payment models to improve access. - **CRISPR Gene-Editing Therapy**: FDA approval of the first CRISPR gene-editing therapy for sickle cell disease, showcasing rapid advancements in gene therapies. - **SPR301 by Spur Therapeutics**: A promising gene therapy candidate for treating Parkinson's disease linked to GBA1 mutations. - **Training Programs for Pharmacists**: Recommendations for establishing training programs for pharmacists and technicians to manage the complexities of high-cost gene therapies. - **InspiroGene by McKesson**: A new business dedicated to supporting the commercialization of cell and gene therapies, enhancing patient access to treatments. - **Value-Based Contracts**: The importance of value-based contracts and insurance coverage for gene therapies is highlighted to improve access and affordability. ### Challenges and Concerns - **High Costs of Therapies**: Gene therapies can be extremely costly, ranging from $400,000 to $2 million per dose, raising concerns about affordability and access. - **High Upfront Costs**: Gene therapies often exceed £1 million per dose, necessitating innovative funding models. - **High Costs of Gene Therapies**: The high upfront costs of gene therapies, such as Bluebird Bio's Lyfgenia and Vertex's Casgevy, pose challenges for patient access and healthcare systems. - **High Costs**: Gene therapies can exceed $3 million, raising concerns about accessibility and affordability for patients. - **Manufacturing Complexities**: Gene therapy manufacturing processes are intricate and costly, leading to challenges in productivity compared to traditional therapies. - **Cost and Accessibility**: High production costs of gene therapies limit patient access, necessitating innovations in manufacturing processes. - **Long-Term Safety**: Concerns regarding long-term safety and potential insertional mutagenesis in gene therapies, despite studies showing promising results. - **Accessibility and Insurance Coverage**: Barriers such as accessibility, insurance coverage, and safety concerns hinder the widespread adoption of gene therapies. - **Labor-Intensive Processes**: The complex nature of gene therapies requires coordination between providers and manufacturers, complicating patient access. - **High Development Costs**: The high costs associated with developing gene therapies pose significant challenges for pharmaceutical companies. - **Regulatory Hurdles**: The evolving regulatory landscape poses challenges for the commercialization and reimbursement of gene therapies. - **Regulatory Complexities**: Despite improvements in the regulatory landscape, challenges remain in navigating the complexities of gene therapy approvals. - **Market Adoption**: Slow uptake of newly approved gene therapies, such as those for sickle cell disease, due to competition with existing treatments. - **Equity in Access**: The potential for inequities in access to gene therapies, particularly for underserved populations affected by conditions like sickle cell disease. - **Manufacturing and Delivery Challenges**: Investment in cell and gene therapy developers has declined due to significant capital requirements for manufacturing. - **Safety of New Technologies**: Ensuring the safety of new gene therapies leads to lengthy regulatory review processes, delaying market entry. - **Regulatory Innovation Needs**: The necessity for tailored regulatory frameworks for individualized therapies remains a challenge. ## Related Topics [[Gene Therapy]]; [[Cell Therapy]]