# Gene Therapy * **Definition:** A medical intervention that involves altering the genes inside a patient's cells to treat or prevent disease, often through the insertion, alteration, or removal of genes. * **Taxonomy:** Healthcare Topics / Gene Therapy ## News * Selected news on the topic of **Gene Therapy**, for healthcare technology leaders * 15.5K news items are in the system for this topic * Posts have been filtered for tech and healthcare-related keywords | Date | Title | Source | | --- | --- | --- | | 5/12/2025 | [**Can Buying CRISPR Therapeutics and Holding It Forever Make You a Millionaire?**](https://www.aol.com/buying-crispr-therapeutics-holding-forever-101800694.html) | [[AOL]] | | 5/7/2025 | [**Apertura Gene Therapy Showcases the Progress of its Novel Human Transferrin Receptor ...**](https://www.prnewswire.com/news-releases/apertura-gene-therapy-showcases-the-progress-of-its-novel-human-transferrin-receptor-aav-capsid-tfr1-capx-and-preclinical-programs-for-tuberous-sclerosis-complex-tsc-at-the-american-society-of-gene-and-cell-therapy-asgct-ann-302448607.html) | [[PR Newswire]] | | 5/5/2025 | [**Gene Therapy Market to Hit US$36.55 Billion by 2032 with 19.4AGR - MarketsandMarkets™**](https://www.globenewswire.com/news-release/2025/05/05/3074053/0/en/Gene-Therapy-Market-to-Hit-US-36-55-Billion-by-2032-with-19-4-CAGR-MarketsandMarkets.html) | [[Globe Newswire]] | | 5/5/2025 | [**Jerapy for Rare, Progressive Vision-Loss Disorder Fails Phase 3 Test**](https://medcitynews.com/2025/05/jnj-gene-therapy-inherited-retinal-rare-disease-vision-loss-xlrp-meiragtx/) | [[MedCity News]] | | 5/5/2025 | [**Gene Therapy Market to Hit US$36.55 Billion by 2032 with 19.4AGR - Yahoo Finance**](https://finance.yahoo.com/news/gene-therapy-market-hit-us-123000543.html) | [[Yahoo Finance]] | | 5/2/2025 | [**Vertex discontinues AAV research for gene therapies - Fierce Biotech**](https://www.fiercebiotech.com/biotech/vertex-discontinues-aav-vector-research-gene-therapies) | [[FierceBiotech]] | | 4/21/2025 | [**Retinitis Pigmentosa Treatment Market Set to Double by 2032, Driven by Gene Therapy Advances**](https://finance.yahoo.com/news/retinitis-pigmentosa-treatment-market-forecast-080100293.html) | [[Yahoo Finance]] | | 3/7/2025 | [**Biopharma Giants Lead Gene Therapy Market Expansion - Latest Developments & Projections**](https://finance.yahoo.com/news/gene-therapy-market-global-forecast-090900277.html) | [[Yahoo Finance]] | | 3/5/2025 | [**Global Cell and Gene Therapy Manufacturing Market to Reach ~USD 10 Billion by 2032 - DelveInsight**](https://www.globenewswire.com/news-release/2025/03/05/3037668/0/en/Global-Cell-and-Gene-Therapy-Manufacturing-Market-to-Reach-USD-10-Billion-by-2032-DelveInsight.html) | [[Globe Newswire]] | | 3/5/2025 | [**Global Cell and Gene Therapy Manufacturing Market to Reach ~USD 10 Billion by 2032**](https://finance.yahoo.com/news/global-cell-gene-therapy-manufacturing-180000731.html) | [[Yahoo Finance]] | | 2/20/2025 | [**Gene Therapy Market to Hit USD 36.55 Billion by 2032 with 19.4AGR - MarketsandMarkets™.**](https://www.prnewswire.com/news-releases/gene-therapy-market-to-hit-usd-36-55-billion-by-2032-with-19-4-cagr--marketsandmarkets-302381166.html) | [[PR Newswire]] | | 2/20/2025 | [**Gene Therapy Market to Hit USD 36.55 Billion by 2032 with 19.4AGR - Yahoo Finance**](https://finance.yahoo.com/news/gene-therapy-market-hit-usd-153000096.html) | [[Yahoo Finance]] | | 2/3/2025 | [**Cancer Gene Therapy Market to Surpass Valuation of US$ 17.57 Billion By 2033 - Astute Analytica**](https://finance.yahoo.com/news/cancer-gene-therapy-market-surpass-123000442.html) | [[Yahoo Finance]] | | 1/21/2025 | [**Asia-Pacific Cell and Gene Therapy Manufacturing QC Research Report 2024-2033 with Danaher, F. Hoffmann-La Roche, and Thermo Fisher Scientific Leading the $1.3+ Billion Market - ResearchAndMarkets.com**](http://www.businesswire.com/news/home/20250121430719/en/Asia-Pacific-Cell-and-Gene-Therapy-Manufacturing-QC-Research-Report-2024-2033-with-Danaher-F.-Hoffmann-La-Roche-and-Thermo-Fisher-Scientific-Leading-the-1.3-Billion-Market---ResearchAndMarkets.com/?feedref=JjAwJuNHiystnCoBq_hl-RLXHJgazfQJNuOVHefdHP-D8R-QU5o2AvY8bhI9uvWSD8DYIYv4TIC1g1u0AKcacnnViVjtb72bOP4-4nHK5ieT3WxPE8m_kWI77F87CseT) | [[Business Wire]] | | 1/3/2025 | [**Cell Therapy and Gene Therapy CDMO Market to Reach USD 11.11 Billion by 2030 - Discover Growth Trends and Insights - Valuates Reports**](https://www.prnewswire.com/news-releases/cell-therapy-and-gene-therapy-cdmo-market-to-reach-usd-11-11-billion-by-2030--discover-growth-trends-and-insights--valuates-reports-302341836.html) | [[PR Newswire]] | | 1/3/2025 | [**Cell Therapy and Gene Therapy CDMO Market to Reach USD 11.11 Billion by 2030 - Yahoo Finance**](https://finance.yahoo.com/news/cell-therapy-gene-therapy-cdmo-151500611.html) | [[Yahoo Finance]] | | 1/2/2025 | [**Gene Therapy Market to Hit Valuation of US$ 42.26 Billion By 2033 - Astute Analytica**](https://finance.yahoo.com/news/gene-therapy-market-hit-valuation-093000249.html) | [[Yahoo Finance]] | | 1/1/2025 | [**Prediction: This Will Be the First Tech Company to Split Its Stock in 2025. (Hint - Yahoo**](https://www.yahoo.com/finance/news/prediction-first-tech-company-split-121100832.html) | [[Yahoo]] | | 12/26/2024 | [**Gene Therapy Market Analysis,Growth, Insights and Future Outlook - Exactitude Consultancy**](https://finance.yahoo.com/news/gene-therapy-market-analysis-growth-121800860.html) | [[Yahoo Finance]] | | 11/29/2024 | [**Cell and Gene Therapy Tools Market to Hit $16.7 Billion by 2029 - Yahoo Finance**](https://finance.yahoo.com/news/cell-gene-therapy-tools-reagents-092400787.html) | [[Yahoo Finance]] | | 11/22/2024 | [**Cancer Gene Therapy Strategic Business Report 2024: Global Market to Grow by $6.5 ...**](https://finance.yahoo.com/news/cancer-gene-therapy-strategic-business-132500106.html) | [[Yahoo Finance]] | | 11/7/2024 | [**Cell And Gene Therapy Market Size to Worth USD 190.82 Billion by 2033 - Straits Research**](https://finance.yahoo.com/news/cell-gene-therapy-market-size-143000986.html) | [[Yahoo Finance]] | | 10/9/2024 | [**Cell and Gene Therapy in Parkinson's Disease Clinical Trial Pipeline Analysis Demonstrates 18+ Key Companies at the Horizon Expected to Transform the Treatment Paradigm, Assesses DelveInsight**](https://www.prnewswire.com/news-releases/cell-and-gene-therapy-in-parkinsons-disease-clinical-trial-pipeline-analysis-demonstrates-18-key-companies-at-the-horizon-expected-to-transform-the-treatment-paradigm-assesses-delveinsight-302270981.html) | [[PR Newswire]] | | 8/28/2024 | [**Gene Therapy for Cardiovascular Disease Set for Exponential Growth by 2031 As Revealed ...**](https://www.whatech.com/og/markets-research/medical/872137-gene-therapy-for-cardiovascular-disease-set-for-exponential-growth-by-2031.html) | whatech.com | | 7/19/2024 | [**Gene Therapy Agreements Analysis Report and Directory 2016-2024: Collaboration, Development, Research and Licensing Deals by Company A-Z, Deal Type and Therapy Area - ResearchAndMarkets.com**](http://www.businesswire.com/news/home/20240719055173/en/Gene-Therapy-Agreements-Analysis-Report-and-Directory-2016-2024-Collaboration-Development-Research-and-Licensing-Deals-by-Company-A-Z-Deal-Type-and-Therapy-Area---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 - **Apertura Gene Therapy**: A company developing gene therapies with a focus on delivering genetic medicines across the blood-brain barrier. - **Genethon**: A non-profit gene therapy R&D organization dedicated to developing therapies for rare genetic diseases. - **Dyno Therapeutics, Inc.**: A company specializing in gene therapy technologies, utilizing AI to create synthetic AAV capsids for improved gene delivery. - **Neurogene**: A biotech company developing gene therapies for rare diseases, including Rett syndrome. - **BridgeBio**: A biotechnology company developing gene therapies, including BBP-812 for Canavan disease, which aims to correct genetic defects. - **Bristol-Myers Squibb**: A global biopharmaceutical company actively evaluating novel gene therapy candidates. - **Spur Therapeutics**: Developing gene therapy candidates for adrenomyeloneuropathy and other conditions. - **Belief BioMed**: A Shanghai-based company with advanced viral vector technologies, partnering with AskBio for gene therapy development. - **Jim Wilson**: Prominent gene therapy researcher and founder of Gemma Biotherapeutics and Franklin Biolabs, focusing on rare disease research and gene therapy services. - **Beijing Tsingke Biotech Co., Ltd.**: A biotech firm that provides gene synthesis services and has showcased its innovations in gene therapy at industry exhibitions. - **Spirovant Sciences**: Introduced SP-101, a novel gene therapy for cystic fibrosis. - **ReNeuron**: A biotechnology company focusing on developing innovative therapies, particularly in the field of gene therapy. - **BridgeBio Pharma**: A company reducing its gene therapy budget and shifting focus after unsatisfactory trial results for its investigational gene therapy. - **NewBiologix**: A company that has launched a platform to enhance the production of viral vectors for gene therapy. - **AskBio**: A company collaborating with Belief BioMed to develop gene therapies targeting diseases with significant unmet medical needs. - **PTC Therapeutics, Inc.**: A biopharmaceutical company that received FDA approval for KEBILIDI, the first gene therapy administered directly to the brain for AADC deficiency. ### Partnerships and Collaborations - **Apertura Gene Therapy and Broad Institute**: Collaboration to develop gene therapy for prion disease, utilizing innovative technologies for effective delivery. - **Apertura Gene Therapy and Rett Syndrome Research Trust**: Collaborating to enhance gene therapy approaches for treating Rett Syndrome. - **Viralgen and Trogenix**: Collaborating to expedite the development of a recombinant adeno-associated virus gene therapy for glioblastoma. - **Hansa Biopharma and Genethon**: Initiated a Phase 2 clinical trial for a gene therapy targeting Crigler-Najjar syndrome. - **AskBio and Belief BioMed**: Strategic collaboration to develop gene therapies utilizing liver-targeted approaches. - **Solid Biosciences and Mayo Clinic**: Collaboration to advance AAV-based gene therapy for genetic cardiomyopathies, with exclusive licenses for cardiac gene therapy programs. - **Children's Hospital of Philadelphia (CHOP)**: Collaborated on a gene therapy trial for Danon disease. - **Genethon and Hansa Biopharma**: Collaborating on a Phase 2 trial of imlifidase for Crigler-Najjar syndrome patients. - **Amarna Therapeutics**: Formed a Scientific Advisory Board to support its gene therapy programs. - **Roche and Dyno Therapeutics**: Roche exercised its option to license a novel capsid for a gene therapy program, resulting in substantial potential earnings for Dyno. - **UAB and Tramontane Therapeutics Inc.**: UAB licensed AAV-FGF21 gene therapy program to Tramontane for treating metabolic diseases. - **CHLA and Keck School of Medicine**: Collaboration to advance gene and cell therapies, supported by significant funding. - **Donaldson and University of Pennsylvania**: Evaluating the scalability of gene therapy product manufacturing to improve accessibility. - **Astellas Pharma and Dyno Therapeutics**: Astellas is collaborating with Dyno to enhance gene delivery methods targeting specific tissues. - **Phenocell and Amarna Therapeutics**: Received a Eurostars grant to validate a novel gene therapy platform for Dry Age-Related Macular Degeneration. - **GEMMA Biotherapeutics and Fiocruz**: Collaboration aimed at advancing research and global access to gene therapies for rare diseases. - **Boston Oncology and King Fahd Medical City**: Collaborating to localize Cell & Gene Therapy in Saudi Arabia, enhancing local medical technologies. - **Teijin Limited and Hilleman Laboratories**: Signed a Memorandum of Understanding to establish a strategic partnership for expanding their CDMO business in cell and gene therapy. ### Innovations, Trends, and Initiatives - **Gene Therapy Market Growth**: Projected to grow from $7.21 billion in 2023 to $36.55 billion by 2032, driven by demand for curative therapies. - **Gene therapy for sickle cell disease**: New approaches are being developed to enhance the efficacy of gene transfer using lentiviral vectors. - **New Gene Therapy for Heart Failure**: A gene therapy developed to reverse heart failure effects by increasing levels of cBIN1 protein, showing significant improvements in animal models. - **Synthetic Super-Enhancer technology**: Trogenix launched a platform aimed at revolutionizing cancer treatment through precision viral immunotherapy. - **AI in Gene Therapy Development**: Genethon is using AI to design new capsids for gene therapy vectors targeting muscle diseases. - **AI in Gene Therapy**: Dyno Therapeutics utilizes AI to improve gene delivery systems, addressing traditional capsid challenges. - **Regulatory Incentives**: Regulatory incentives like orphan drug designations and accelerated approvals are promoting gene therapy development. - **Self-amplifying mRNA and lipid nanoparticle technologies**: CSL Behring is focusing on these promising areas of gene therapy. - **SP-101 for Cystic Fibrosis**: A novel gene therapy showing effective delivery and long-term effects in animal models. - **Regulatory Advances**: Apertura Gene Therapy received FDA feedback endorsing a streamlined preclinical development strategy for its AAV capsid. - **CRISPR-Cas9 technology**: Advancements in gene editing technology are significantly enhancing cancer gene therapy. - **AAV Gene Therapy**: Advancements in adeno-associated virus gene therapy are making strides in treating conditions like spinal muscular atrophy. - **ABBV-RGX-314**: A novel gene therapy for treating neovascular age-related macular degeneration (AMD) that significantly reduces treatment burden. - **Machine Learning in Gene Therapy**: A study introduced a model to predict the fitness of AAV capsids, potentially reducing the need for extensive in vitro experiments. - **CRISPR/Cas9**: A gene-editing tool facilitating precise genome editing, enhancing research and development in gene therapy. - **Market Growth**: The global gene therapy market is projected to reach $6.0 billion by 2030, growing at a CAGR of 23.1% from $1.4 billion in 2023. - **Oncolytic Virotherapy**: A key segment of cancer gene therapy that generated significant revenue, indicating a shift towards innovative treatment approaches. - **AI in Capsid Engineering**: Emerging AI-based methods are enhancing the optimization of gene therapy vectors. ### Challenges and Concerns - **Limited Treatment Reach**: Despite advancements, the accessibility of gene therapies remains a significant challenge. - **High Development Costs**: Gene therapy development is expensive, with significant costs associated with clinical trials and manufacturing. - **Immune reactions and long-term effects**: Gene therapy faces challenges such as immune responses to therapies and the long-term effects of gene modifications. - **Adverse Events in Trials**: Recent studies have shown treatment-related adverse events in gene therapy trials, raising safety concerns. - **Regulatory Hurdles**: Gene therapy faces significant regulatory challenges that can impede development and approval processes. - **Ethical concerns and treatment costs**: High costs and ethical considerations surrounding gene editing and therapy limit accessibility for many patients. - **Infrastructure Needs**: There is a need for improved infrastructure and expertise in various regions to support gene therapy advancements. - **Declining Investment**: A notable decrease in early investment in gene therapy, with funding dropping significantly from previous years. - **Regulatory Changes**: Upcoming regulatory changes in Europe may undervalue gene therapy products, limiting patient access. - **COVID-19 Impact**: The pandemic adversely affected the gene therapy industry, particularly in cardiovascular disease, due to site limitations and supply chain disruptions. - **Complexity of Trials**: Gene therapy trials face unique complexities such as patient heterogeneity and safety assessments, complicating logistics and planning. - **High Costs and Accessibility**: High investment costs, a shortage of skilled professionals, and limited insurance coverage may hinder gene therapy expansion. - **Measurement Technique Issues**: Concerns about the accuracy and reliability of popular measurement methods like PCR-ELISA in gene therapy. - **High Treatment Costs**: The high costs associated with gene therapies pose significant barriers to patient access. - **Safety Concerns**: Concerns regarding the potential carcinogenicity of gene therapies and the need for transparent data on vector genotoxicity. - **Access Issues**: Many patients worldwide face geographical and eligibility constraints that limit access to gene therapies. - **High Costs**: The high costs associated with patented gene therapies limit treatment reach, particularly in developing regions. ## Related Topics [[Gene Therapies]]; [[Cell Therapy]]; [[Gene Editing]]