https://www.ucl.ac.uk/news/2025/dec/world-first-base-edited-gene-therapy-helps-patients-fight-previously-incurable-blood-cancer
A groundbreaking new treatment using genome-edited immune cells, developed by scientists at UCL and Great Ormond Street Hospital (GOSH), has shown promising...
gene therapyworldfirstbaseedited
https://www.chop.edu/news/worlds-first-patient-treated-personalized-crispr-gene-editing-therapy-childrens-hospital
Landmark study from CHOP and Penn Medicine showcases the power of customized gene editing therapy to treat patient with rare metabolic disease.
worldfirstpatienttreatedpersonalized
https://www.stanfordchildrens.org/en/about/news/releases/2026/dystrophic-epidermolysis-bullosa.html
Physicians at Stanford Medicine Children’s Health have achieved a breakthrough in the treatment of epidermolysis bullosa (EB) through a pioneering surgical...
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https://www.merative.com/blog/veristat-gene-therapy
Learn how Veristat, the global Science-First™ CRO, reduced gene therapy trial costs thanks to efficient patient data management with Zelta.
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https://www.streetwisereports.com/article/2025/10/09/biotech-firm-uncovers-breakthrough-gene-therapy-in-new-york.html
Lexeo Therapeutics Inc. (LXEO:NASDAQ) advanced its LX2006 program after new FDA feedback supported an accelerated approval path. Read how interim data showed...
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https://www.insideprecisionmedicine.com/topics/precision-medicine/make-waves-inside-the-gene-therapy-bringing-natural-hearing-to-deaf-children/
Oct 17, 2025 - Regeneron's experimental dual-AAV gene therapy improved hearing and speech perception in otoferlin-deficient children.
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https://medlineplus.gov/genesandgenetherapy.html
Genes are the building blocks of inheritance. Read more about genes and the currently experimental technique of gene therapy.
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https://www.genethon.com/long-term-success-of-gene-therapy-in-patients-with-fanconi-anemia/
Dec 17, 2024 - The results of a clinical trial conducted by Rocket Pharmaceuticals and the Spanish CIEMAT teams, in collaboration with Genethon, which helped design and...
long termgene therapysuccesspatients
https://www.biomerieux.com/corp/en/our-offer/pharmaceutical-quality-control/cell-and-gene-therapies.html
Navigating the complexities of manufacturing cell and gene therapies while ensuring their quality, safety, and efficacy is crucial. Discover how our advanced...
gene therapycellampsolutionspioneering
https://thegivingblock.com/donate/alliance-for-cancer-gene-therapy/
Jan 9, 2025 - Donate Bitcoin to nonprofits like Alliance for Cancer Gene Therapy who accept cryptocurrency, crypto donations are tax deductible.
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https://resilience.com/blog/overcoming-gene-therapy-obstacles
Sep 9, 2025 - Gene therapy and viral vector manufacturing have yet to achieve the level of standardization that typifies established modalities.
overcoming obstaclesgene therapymanufacturing
https://www.bioworld.com/articles/725024-trogenix-raises-70m-series-a-for-glioblastoma-gene-therapy-trial?v=preview
Newco Trogenix Ltd. has emerged from incubation and raised £70 million (US$94.1 million) in a series A, as it prepares the ground for a U.S/U.K. clinical...
gene therapyraisesseriesglioblastomatrial
https://www.clinicaltrialsarena.com/sponsored/pushing-boundaries-in-cell-and-gene-therapy/
Nov 27, 2025 - TriLink BioTechnologies senior scientist Dr Cody Palumbo discusses the blockers to manufacturing mRNA-based gene editing therapies.
nextactmrnapushingboundaries
https://www.solventum.com/en-us/home/purification-filtration/biopharmaceutical-purification/gene-therapy/
Intensify your gene therapy bioprocessing, reduce costs and speed the path from discovery to market by eliminating endonuclease treatment in the clarification...
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https://www.biaseparations.com/sl/event_items/cell-gene-therapy-international-eu/
Nov 11, 2025 - Listen to our presentation Speaker: Aleš Štrancar, Managing Director, Sartorius BIA Separations Date and Time: Monday, December 1, 2025 at 15:00 PM (Track...
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https://www.genethon.com/our-science/the-future-of-gene-therapy/understanding-gene-therapy/
Dec 19, 2024 - Gene therapy is a set of techniques that involve correcting the function of a “sick” gene, often by replacing it with a functional gene (“drug gene”)....
gene therapyunderstanding
https://www.drugdiscoverytrends.com/prominent-cell-and-gene-therapy-vendors-in-2023/
Jul 26, 2023 - Explore a curated list of notable cell and gene therapy vendors and suppliers. Discover their focus areas, partnerships and focus areas.
gene therapyvendorssuppliersgrowingcell
https://www.bioworld.com/articles/728034-adolores-ca8-gene-therapy-designated-orphan-drug?v=preview
Adolore Biotherapeutics Inc. has announced that the FDA has granted orphan drug designation to the company’s Kv7-activating rdHSV-CA8* gene therapy for...
gene therapydesignatedorphandrugbioworld
https://www.biomerieux.com/corp/en/blog/pharmaceutical-qc/accelerating-the-pace-of-innovation-in-cell-and-gene-therapy.html
With the introduction of new guidances and the endorsement of USP has not only provided a clear support for rapid microbiological methods, but it has also...
gene therapyacceleratingpaceinnovationcell
https://www.statnews.com/2025/08/21/sarepta-gene-therapy-families-left-behind/
Oct 10, 2025 - After deaths and FDA setbacks, Sarepta abandoned LGMD gene therapy, leaving families desperate and patients without long-promised treatments.
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https://www.biopharmadive.com/news/pfizer-duchenne-gene-therapy-phase-3-sarepta/592936/
Sarepta, though, may try to seek accelerated approval using Phase 2 data that's due imminently, along with results from another, smaller trial.
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https://www.drugdiscoverytrends.com/100-top-cell-gene-therapy-companies-watch-2023/
Dec 20, 2024 - Discover the top 100 companies leading the way in the promising field of cell and gene therapy, including public and private companies.
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https://www.genethon.com/esgct-2025-genethon-at-the-forefront-of-gene-therapy/
Oct 2, 2025 - From October 7 to 10, 2025, Seville will host the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), the unmissable gathering of...
gene therapy
https://www.biomerieux.com/us/en/our-offer/pharmaceutical-quality-control/cell-and-gene-therapies.html
Navigating the complexities of manufacturing cell and gene therapies while ensuring their quality, safety, and efficacy is crucial. Discover how our advanced...
gene therapycellampsolutionspioneering
https://www.livescience.com/health/from-gene-therapy-breakthroughs-to-preventable-disease-outbreaks-the-health-trends-that-will-shape-2026
Live Science's health channel editor makes predictions about the medical breakthroughs and public health shifts to come in 2026.
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https://www.thermofisher.com/us/en/home/clinical/cell-gene-therapy/gene-therapy.html
Our end-to-end solutions, high-quality products and unrivaled services support you from gene therapy development and research to commercialization.
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https://www.statnews.com/2025/11/18/fda-warning-on-sarepta-gene-therapy-for-muscular-dystrophy/
Nov 18, 2025 - Risk of “acute, serious, and life-threatening” heart inflammation is now on the label of Sarepta's gene therapy for Duchenne muscular dystrophy.
gene therapynewwarningsarepta
https://adultgamescollector.com/2025/12/21/gene-therapy-root/
Date: 21.12.2025 Language: English Version: 0.11 Censored: No Size: 4120 mb Tags: HTML, Sandbox, Text-Based, Erotic Adventure, Male Protagonist, Sexy Girls,...
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https://penntoday.upenn.edu/news/penn-medicine-gene-therapy-switch-may-offer-non-addictive-pain-relief
In a preclinical study, Penn Medicine researchers have uncovered a new gene therapy that targets only pain signals while leaving the rest of the brain...
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https://www.livescience.com/health/medicine-drugs/groundbreaking-gene-therapy-is-first-treatment-for-huntingtons-disease-to-slow-the-condition
Results from a three-year trial suggest an experimental gene therapy for Huntington's disease can slow the progression of the deadly condition by 75%.
gene therapygroundbreakingfirsttreatmenthuntington
https://www.khaleejtimes.com/kt-network/fakeeh-university-hospital-sets-new-benchmarks-in-advanced-gene-therapy
Discover how Fakeeh University Hospital is setting new benchmarks in advanced gene therapy, offering cutting-edge treatments for rare genetic disorders
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https://www.patheon.com/us/en/our-capabilities/advanced-therapies.html?utm_campaign=2024-psg-general&utm_campaignchild=end-to-end&utm_specialty=end2end&utm_campaignregion=global&utm_campaigncountry=&utm_source=thermofisher-brandpage&utm_medium=referral&utm_content=our-capabilities-advanced-therapies&utm_campaignassettype=web-page&utm_term=
We are an experienced global CDMO partner offering development and manufacturing services for viral vectors, plasmid DNA, cell therapies, and mRNA therapeutics...
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https://www.bioworld.com/articles/728939-apertura-gene-therapy-signs-frrs1l-license-agreement?v=preview
Nonprofit Finding Hope for Frizzle (FRRS1L) and Apertura Gene Therapy have signed a license agreement for the development of a gene therapy for FRRS1L disease,...
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https://www.gosh.nhs.uk/news/ready-made-t-cell-gene-therapy-tackles-incurable-t-cell-leukaemia/
A groundbreaking new treatment using gene-edited immune cells, developed by scientists at Great Ormond Street Hospital (GOSH) and University College London...
gene therapycelltackles
https://primaryimmune.org/understanding-primary-immunodeficiency/treatment/gene-therapy
Gene therapy is still in clinical trials for several types of primary immunodeficiency (PI), including severe combined immunodeficiency (SCID), chronic...
gene therapyimmune deficiencyfoundation
https://www.ucl.ac.uk/news/2025/nov/boy-given-world-first-gene-therapy-made-ucl-scientists-thriving
A young boy born with a devastating, rare genetic condition has been given a new lease of life thanks to a team of UCL scientists who manufactured a pioneering...
gene therapyboygivenworldfirst
https://piyarishayari.com/rewriting-the-code-of-aging-gene-therapy-in-later-life/
Jul 29, 2025 - Aging is something we all experience, and for many seniors, staying healthy and active is a top priority.
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https://www.statnews.com/2025/06/19/sarepta-therapeutics-duchenne-muscular-dystrophy-company/
Jun 19, 2025 - With a tragedy in the news — the death of a second child tied to the therapy — the company faces market consequences, too.
gene therapynextsareptatherapeutics
https://www.statnews.com/2025/06/18/top-gene-therapy-regulator-forced-out-at-fda/
Jun 19, 2025 - She had worked closely with former CBER head Peter Marks on establishing new paths for bringing gene therapies to market, particularly in rare diseases.
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https://www.biopharmadive.com/news/harvard-gene-therapy-dyno-sarepta-novartis/577675/
The startup announced collaborations with Novartis and Sarepta, both leading gene therapy makers, that could be worth $2 billion in total.
dynospinsharvardlaboratories
https://www.biopharmadive.com/news/could-novartis-gene-therapy-have-more-than-one-price/555453/
Indication-based pricing may be possible for less severe patients, who would receive a smaller dose. Lower manufacturing costs would lessen the blow to...
gene therapycouldnovartisoneprice
https://mgriblog.org/2025/06/02/race-to-save-a-life-how-an-act-of-collaboration-led-to-a-bespoke-gene-therapy-treatment/
Jun 2, 2025 - By sharing a newly designed enzyme for gene editing, Mass General Brigham researchers helped to develop a life-saving gene therapy treatment.
racesavelifeactcollaboration
https://www.vitadao.com/blog/vitarna-q4-progress-first-tokenized-gene-therapy-scales-to-production-2025-eyes-animal-studies
In a significant update on October 24, 2024, VitaRNA/Artan Bio showcased continued advancement in their groundbreaking approach to treating genetic diseases...
gene therapyprogressfirsttokenizedscales
https://www.agcbio.com/news/agc-biologics-approved-to-manufacture-first-fda-approved-gene-therapy
ACG Biologics Milan site has clearances from both U.S. and European regulators to supply patients with HSC-based gene therapy
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https://mdaquest.org/say-yes-to-gene-therapy-factors-to-consider-when-gene-therapy-is-an-option/
Nov 14, 2025 - When you have access to gene therapy for a neuromuscular disease, weigh the risks and benefits for yourself or your child in your decision-making.
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https://www.redcrossblood.org/biomedical-services/specialty-services/cell-and-gene-therapy-solutions.html
The Red Cross provides cell and gene therapy source materials and collection services to support both clinical trials and commercial therapies. Get more...
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https://www.statnews.com/2026/01/15/regeneron-george-yancopoulos-jpm-biotech/
Jan 15, 2026 - At the JP Morgan Healthcare Conference, the longtime biotech executive was, as usual, outspoken in an interview with STAT.
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https://www.biomerieux.com/us/en/our-offer/pharmaceutical-quality-control/cell-and-gene-therapy.html
Cutting-edge cellular therapy products demand state-of-the-art quality control.
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https://www.bioworld.com/articles/728904-anti-tfr1-nanobody-i2s-gene-therapy-corrects-hunter-syndrome-neuropathy-in-mice?v=preview
Hunter syndrome, also called mucopolysaccharidosis II, is an X-linked genetic lysosomal disorder caused by loss-of-function mutations in the IDS gene, encoding...
gene therapyanticorrectshuntersyndrome
https://www.genethon.com/
Sep 11, 2025 - Genethon develops innovative therapies for rare diseases of the eye, the liver, the immune system and for neuromuscular diseases.
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https://www.statnews.com/2025/06/19/readout-loud-podcast-fda-sarepta-verdun-gene-therapy-harvard/
Jun 19, 2025 - STAT reporters discuss more turmoil in the FDA's regulation of gene therapies and a model of funding academic research with private capital.
gene therapyloudpodcastfdaturmoil
https://www.statnews.com/2025/12/15/gene-therapy-capsida-biotherapeutics-clinical-trial-death-ramifications/
Dec 16, 2025 - Researchers fear a broad risk for any virus genetically designed to penetrate the blood-brain barrier after no hint of issues prior to Capsida trial death.
gene therapytrialdeathpromisingarea
https://www.insideprecisionmedicine.com/topics/precision-medicine/reporters-notebook-the-overdue-breakout-for-gene-therapy-and-gene-editing/
Oct 21, 2025 - At the 2025 Meeting on the Mesa, innovators reclaim their focus, discipline, and cautiously optimistic momentum after years of hype and hard lessons.
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https://cca-reports.ca/events/breaking-through/
Nov 6, 2025 - Breaking Through will explore the past, present, and future of somatic gene therapy research and...
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https://www.the-scientist.com/category/health/cell-and-gene-therapy
The Scientist's content categorized under the health category: Cell & Gene Therapy.
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https://jaguargenetherapy.com/
Mar 14, 2025 - At Jaguar Gene Therapy, we believe in a world where patients and families won’t have to hear those words. Our mission is to accelerate breakthroughs in gene...
gene therapyjaguaracceleratingbreakthroughs
https://www.ppd.com/therapeutic-expertise/cell-and-gene-therapy-cro/
Aug 15, 2025 - Working with a cell and gene therapy CRO across the development lifecycle fosters better integration of services and capabilities throughout your ATMP clinical...
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https://tactix.com/news/cell-and-gene-therapy-expert-and-entrepreneur-dana-hammill-joins-tactix-team-as-life-sciences-advisor/
Jan 24, 2023 - Tactix Real Estate Advisors A Unique brokerage, exclusively for tenants
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https://www.genezen.com/insights/serving-patients-a-shared-mission-in-rare-disease-gene-therapy/
Nov 17, 2025 - At Genezen, our mission has always been rooted in a singular goal—serving patients. That ethos came to life during a recent panel discussion led by...
rare diseasegene therapyservingpatientsinnovation
https://hackaday.com/2025/11/11/gene-therapy-aims-to-slow-huntingtons-disease-to-a-crawl/
Despite the best efforts of modern medicine, Huntington’s disease is a condition that still comes with a tragic prognosis. Primarily an inherited...
gene therapyaimsslowdiseasecrawl
https://biobridgeglobal.org/uncategorized/progress-when-systems-stand-still-resilience-and-access-in-cell-and-gene-therapy/
Nov 10, 2025 - Read to explore how resilience, access, and innovation drive progress in cell and gene therapy—even amid challenges like government shutdowns.
progresssystemsstandstillresilience
https://pharmacy.ufl.edu/2025/07/31/nih-award-supports-new-extracellular-vesicle-loading-platform-to-develop-gene-therapy/
Dr. Mei He’s team will collaborate with experts from the UF Gene Therapy Center and the UF CTSI Good Manufacturing Practice Facility.
nihawardsupportsnewloading
https://www.synconaltd.com/news-insights/news/spur-therapeutics-presents-updated-phase-1-2-data-on-its-gene-therapy-candidate-in-gaucher-disease-at-esgct-32nd-annual-congress/
spurtherapeuticspresentsupdatedphase
https://european-biotechnology.com/sponsored-publications/innovative-approaches-to-gene-therapy/
Oct 16, 2025 - Watch this expert-led session to explore the latest trends and insights in gene therapy, including how to model immunosuppressive regimens, key study-design...
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https://www.statnews.com/2025/09/10/child-dies-after-receiving-new-experimental-gene-therapy/
Sep 11, 2025 - The treatment by Capsida Biotherapeutics was designed to treat STXBP1 encephalopathy
gene therapychilddiesreceivingnew
https://www.livescience.com/health/genetics/in-a-first-congenital-deafness-in-teens-and-adults-treated-with-new-gene-therapy
This is the first time such results have been achieved in both children and adult patients born with a specific type of congenital deafness.
firstcongenitaldeafnessteensadults
https://www.iflscience.com/radical-new-treatment-clears-disease-in-64-percent-of-patients-with-incurable-cancer-81836
Some of the early patients in the trial are still free of cancer after three years.
gene therapyworldfirstclearsdisease
https://www.hklaw.com/en/services/practices/life-sciences/cell-and-gene-therapy
Holland & Knight's Cell and Gene Therapy team guides clients through legislative and regulatory challenges to help breakthrough products gain FDA...
gene therapycellpracticeshollandamp
https://njbmagazine.com/njb-news-now/njbiofutures-launches-to-develop-cell-and-gene-therapy-workforce/
Feb 5, 2025 - The New Jersey Council of County Colleges today launched NJBioFutures, a workforce development coalition aimed at creating a sustainable
gene therapylaunchesdevelopcellworkforce
https://news.ufl.edu/2024/09/blindness-gene-therapy/
Patients born with a certain genetic blindness experienced the world in an entirely new way after a single treatment with gene therapy developed at UF.
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https://www.aabb.org/education-career/education/calendar/event/2026/08/12/aabb-ecast/optimizing-apheresis-starting-material-collections-for-stem-cell-gene-therapy-manufacturing-for-patients-with-genetic-hemoglobinopathies-26el-662
This program will provide an overview of gene therapy for hemoglobinopathies like sickle cell disease and beta-thalassemia illustrating gene editing strategies.
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https://www.thermofisher.com/us/en/home/clinical/cell-gene-therapy.html
From discovery to commercialization, we've got you covered. Connect to cell and gene therapy solutions, capabilities, and guidance for your therapeutics.
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https://www.bbc.co.uk/news/videos/ckg90exve0vo
Toddler Oliver and his dad Ricky join Jon and Sally on the Breakfast sofa, to discuss his progress after his ground-breaking gene therapy.
oliverthreewonderfulworldfirst
https://www.biopharmadive.com/news/fda-reject-ultragenyx-gene-therapy-complete-response/752920/
While the CRL derails a near-term clearance, one analyst described the rejection as more of “a speed bump to approval, rather than a roadblock.”
gene therapybiopharma divefdarejectsmanufacturing
https://dedhamgroup.com/how-healthcare-systems-can-support-cell-and-gene-therapy-growth/
Feb 27, 2025 - The ability of healthcare systems to support CGT growth depends on a range of internal and external factors.
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https://www.clinicaltrialsarena.com/news/zhongmou-therapeutics-rp-gene-therapy-shows-promise-in-first-in-human-trial/
Nov 27, 2025 - Zhongmou’s gene therapy has shown promise in treating the symptoms of retinitis pigmentosa (RP) in a single-site trial in China.
gene therapyrpshowspromisefirst
https://researchtriangle.org/industries/life-sciences/gene-cell-therapy/
Jun 2, 2025 - With three top-tier research institutions, nationally acclaimed hospitals, and a highly educated workforce, the Research Triangle Region has everything you...
cell therapygeneampresearchtriangle
https://www.statnews.com/2025/09/24/huntingtons-gene-therapy-uniqure/
Sep 24, 2025 - Results are likely to support the first approval of a genetic treatment for the rare neurodegenerative condition.
gene therapysloweddiseaseprogressionkey
https://www.patheon.com/us/en/our-capabilities/advanced-therapies.html
We are an experienced global CDMO partner offering development and manufacturing services for viral vectors, plasmid DNA, cell therapies, and mRNA therapeutics...
gene therapycdmo servicesendcellpatheon
https://www.statnews.com/2024/12/09/pirovolakis-gene-therapy-rare-disease-spg50-lockard/
Dec 26, 2024 - Terry Pirovolakis built a gene therapy to save his son from a rare disease. He has become the first stop and last hope for other desperate parents.
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https://www.pharmaceutical-technology.com/data-insights/innovators-cell-gene-therapy-recombinant-aav-gene-therapy-pharmaceutical/
Jul 29, 2024 - GlobalData uncovers the leading innovators in recombinant AAV gene therapy for the pharmaceutical industry.
leading innovatorsgene therapy
https://www.statnews.com/topic/gene-therapy/
Stay up to date on the latest news and advances in gene editing, including CAR-T cell therapy, stem cell research, and the allure of regenerative medicine.
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https://njbmagazine.com/njb-news-now/novartis-receives-fda-approval-for-spinal-muscular-atrophy-gene-replacement-therapy/
Nov 26, 2025 - The FDA has approved Itvisma for the treatment of children two years and older, teens and adults living with spinal muscular atrophy
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https://www.statnews.com/2025/10/10/sarepta-gene-therapy-limb-girdle-muscular-dystrophy/
Oct 10, 2025 - Sarepta says it has what could be the first gene therapy for limb girdle muscular dystrophy. But getting through the FDA may be tough.
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https://www.cryoport.com/applications/gene-therapy/
Dec 1, 2025 - Cryoport Elite® smart shippers are scientifically engineered to provide increased security for irreplaceable commodities. Learn more.
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https://www.biopharmadive.com/news/ucb-gene-therapy-deal-lacerta-handl/588915/
With patent expirations nearing for a top-selling drug, the Belgian pharma will buy one company and partner with another to add several early-stage projects in...
gene therapyucbstrikesdealsbuild
https://www.genezen.com/genezen-announces-manufacturing-partnership-with-humane-genomics-to-advance-oncolytic-virus-therapy-for-pediatric-liver-cancer/
Nov 12, 2025 - Genezen, a leading gene therapy CDMO, has partnered with Elly’s Team, a foundation dedicated to accelerating the translation of medical research into...
partnersteamdevelopment