# sources

node_name: Gene Therapy Durability Uncertainty
created_at: 2026-04-19 20:11:05
url:
https://www.fiercepharma.com/pharma/biomarin-officially-pulls-plug-hemophilia-gene-therapy-roctavian-taking-119m-write-after

title:
fiercepharma.com: Biomarin officially pulls plug hemophilia gene therapy roctavian taking 119m write after


title: biospace.com: Sickle cell gene therapies casgevy and lyfgenia still lacking traction 2 years in
node_name: Gene Therapy One-Time Cost Reimbursement Crisis
created_at: 2026-04-19 20:11:05
url:
https://www.biospace.com/drug-development/sickle-cell-gene-therapies-casgevy-and-lyfgenia-still-lacking-traction-2-years-in


title: chop.edu: Worlds first patient treated personalized crispr gene editing therapy childrens hospital
node_name: Personalized In Vivo CRISPR Therapy (KJ Muldoon)
created_at: 2026-04-19 20:11:05
url:
https://www.chop.edu/news/worlds-first-patient-treated-personalized-crispr-gene-editing-therapy-childrens-hospital


node_name: Personalized In Vivo CRISPR Therapy (KJ Muldoon)
created_at: 2026-04-19 20:11:05
url:
https://www.nih.gov/news-events/news-releases/infant-rare-incurable-disease-first-successfully-receive-personalized-gene-therapy-treatment

title:
nih.gov: Infant rare incurable disease first successfully receive personalized gene therapy treatment


title: progen.com: Crispr cas meets aav navigating the complexities of gene editing delivery
node_name: AAV Vector Immunogenicity Exclusion Problem
created_at: 2026-04-19 20:11:05
url:
https://www.progen.com/post/crispr-cas-meets-aav-navigating-the-complexities-of-gene-editing-delivery


node_name: LNP Liver-Targeted Gene Delivery Platform
created_at: 2026-04-19 20:11:05
url:
https://crisprtx.com/about-us/press-releases-and-presentations/crispr-therapeutics-provides-first-quarter-2025-financial-results-and-announces-positive-top-line-data-from-phase-1-clinical-trial-of-ctx310-targeting-angptl3

title:
crisprtx.com: Crispr therapeutics provides first quarter 2025 financial results and announces positive top line data from phase 1 clinical trial of ctx310 targeting angptl3


title: aafp.org: Will the high price of gene therapy for sickle cell disease put this cure out of reach
node_name: Myeloablative Conditioning Barrier to Gene Therapy Uptake
created_at: 2026-04-19 20:11:05
url:
https://www.aafp.org/pubs/afp/afp-community-blog/entry/will-the-high-price-of-gene-therapy-for-sickle-cell-disease-put-this-cure-out-of-reach.html


title: ir.crisprtx.com: Crispr therapeutics provides business update and reports third 6
node_name: CRISPR Cardiovascular Horizontal Expansion
created_at: 2026-04-19 20:11:05
url:
https://ir.crisprtx.com/news-releases/news-release-details/crispr-therapeutics-provides-business-update-and-reports-third-6/


node_name: In Vivo Cas9 Immune Hepatotoxicity Mechanism
created_at: 2026-04-19 20:11:05
url:
https://www.cgtlive.com/view/intellia-phase-3-trials-transthyretin-amyloidosis-gene-editing-therapy-nex-z-hold-grade-4-liver-ae

title:
cgtlive.com: Intellia phase 3 trials transthyretin amyloidosis gene editing therapy nex z hold grade 4 liver ae


node_name: In Vivo Cas9 Immune Hepatotoxicity Mechanism
created_at: 2026-04-19 20:11:05
url:
https://www.insideprecisionmedicine.com/topics/precision-medicine/reporters-notebook-is-intellias-patient-death-the-nail-in-the-coffin-for-cas9/

title:
insideprecisionmedicine.com: Reporters notebook is intellias patient death the nail in the coffin for cas9


title: ir.intelliatx.com: Intellia therapeutics presents positive longer term phase 1 data
node_name: ATTR Amyloidosis CRISPR Common Disease Pivot
created_at: 2026-04-19 20:11:05
url:
https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-presents-positive-longer-term-phase-1-data


title: technologyreview.com: The first crispr cure might kickstart the next big patent battle
node_name: CRISPR-Cas9 IP Bifurcation: Broad vs Berkeley
created_at: 2026-04-19 20:11:05
url:
https://www.technologyreview.com/2023/12/01/1084152/the-first-crispr-cure-might-kickstart-the-next-big-patent-battle/


title: health.harvard.edu: Gene editing a one time fix for dangerously high cholesterol
node_name: siRNA RNAi Liver Therapy as CRISPR Competitive Floor
created_at: 2026-04-19 20:11:05
url:
https://www.health.harvard.edu/heart-health/gene-editing-a-one-time-fix-for-dangerously-high-cholesterol


node_name: Hemophilia Gene Therapy Market Collapse
created_at: 2026-04-19 20:11:05
url:
https://www.managedhealthcareexecutive.com/view/gene-therapy-may-be-a-once-in-lifetime-treatment-that-gives-some-patients-and-providers-pause-and-a-willingness-to-wait-it-out-until-a-better-therapy-comes-along-amcp-annual-2026

title:
managedhealthcareexecutive.com: Gene therapy may be a once in lifetime treatment that gives some patients and providers pause and a willingness to wait it out until a better therapy comes along amcp annual 2026


node_name: Elevidys AAVrh74 DMD Platform Shutdown
created_at: 2026-04-19 20:11:05
url:
https://www.fda.gov/news-events/press-announcements/fda-requests-sarepta-therapeutics-suspend-distribution-elevidys-and-places-clinical-trials-hold

title:
fda.gov: Fda requests sarepta therapeutics suspend distribution elevidys and places clinical trials hold


title: chemistryworld.com: 4021935
node_name: Elevidys AAVrh74 DMD Platform Shutdown
created_at: 2026-04-19 20:11:05
url:
https://www.chemistryworld.com/news/sarepta-gene-therapy-deaths-highlight-tragic-rare-disease-dilemma/4021935.article


title: ir.wavelifesciences.com: Wave life sciences announces positive update ongoing
node_name: ADAR RNA Editing: Reversible Therapeutic Gene Correction
created_at: 2026-04-19 20:11:05
url:
https://ir.wavelifesciences.com/news-releases/news-release-details/wave-life-sciences-announces-positive-update-ongoing


title: vervetx.gcs-web.com: Lilly acquire verve therapeutics advance one time treatments
node_name: Verve/Eli Lilly PCSK9 Base Editing Acquisition
created_at: 2026-04-19 20:11:05
url:
https://vervetx.gcs-web.com/news-releases/news-release-details/lilly-acquire-verve-therapeutics-advance-one-time-treatments


node_name: Verve/Eli Lilly PCSK9 Base Editing Acquisition
created_at: 2026-04-19 20:11:05
url:
https://www.biopharminternational.com/view/lilly-to-acquire-verve-therapeutics-in-deal-targeting-one-time-gene-editing-treatments-for-cardiovascular-disease

title:
biopharminternational.com: Lilly to acquire verve therapeutics in deal targeting one time gene editing treatments for cardiovascular disease


title: vervetx.gcs-web.com: Verve therapeutics announces positive initial data heart 2 phase
node_name: Verve/Eli Lilly PCSK9 Base Editing Acquisition
created_at: 2026-04-19 20:11:05
url:
https://vervetx.gcs-web.com/news-releases/news-release-details/verve-therapeutics-announces-positive-initial-data-heart-2-phase/


title: cms.gov: Cms expands access lifesaving gene therapies through innovative state agreements
node_name: CMS Outcomes-Based CGT Payment Innovation
created_at: 2026-04-19 20:11:05
url:
https://www.cms.gov/newsroom/press-releases/cms-expands-access-lifesaving-gene-therapies-through-innovative-state-agreements


title: manatt.com: Cms announces 33 states participating in cmmi cgt access model
node_name: CMS Outcomes-Based CGT Payment Innovation
created_at: 2026-04-19 20:11:05
url:
https://www.manatt.com/insights/insight/cms-announces-33-states-participating-in-cmmi-cgt-access-model


title: advisory.avalerehealth.com: Understanding the cms cell and gene therapy access model
node_name: CMS Outcomes-Based CGT Payment Innovation
created_at: 2026-04-19 20:11:05
url:
https://advisory.avalerehealth.com/insights/understanding-the-cms-cell-and-gene-therapy-access-model


title: rolandberger.com: Cutting the cost of gene therapy manufacturing
node_name: AAV Manufacturing Cost-to-Price Disconnect
created_at: 2026-04-19 20:11:05
url:
https://www.rolandberger.com/en/Insights/Publications/Cutting-the-cost-of-gene-therapy-manufacturing.html


node_name: AAV Manufacturing Cost-to-Price Disconnect
created_at: 2026-04-19 20:11:05
url:
https://www.insights.bio/cell-and-gene-therapy-insights/journal/article/3585/the-aav-cdmo-market-in-august-2025-navigating-the-crossroads-of-capacity-complexity-and-cost

title:
insights.bio: The aav cdmo market in august 2025 navigating the crossroads of capacity complexity and cost


title: genengnews.com: Asgct 2025 worlds first patient treated with personalized crispr therapy
node_name: Base Editing Clinical Breakthrough
created_at: 2026-04-19 20:11:05
url:
https://www.genengnews.com/topics/genome-editing/asgct-2025-worlds-first-patient-treated-with-personalized-crispr-therapy/


node_name: N-of-1 Bespoke CRISPR FDA Paradigm
created_at: 2026-04-19 20:11:05
url:
https://www.chop.edu/news/childrens-hospital-philadelphia-marks-one-year-anniversary-worlds-first-personalized-crispr

title:
chop.edu: Childrens hospital philadelphia marks one year anniversary worlds first personalized crispr


title: statnews.com: Vertex crispr sickle cell treatment casgevy faces rollout bottleneck
node_name: CD34+ Stem Cell Collection Yield Constraint
created_at: 2026-04-19 20:11:05
url:
https://www.statnews.com/2026/02/05/vertex-crispr-sickle-cell-treatment-casgevy-faces-rollout-bottleneck/


node_name: Zolgensma SMA Presymptomatic Treatment Model
created_at: 2026-04-19 20:11:05
url:
https://www.novartis.com/news/media-releases/novartis-shares-zolgensma-long-term-data-demonstrating-sustained-durability-75-years-post-dosing-100-achievement-all-assessed-milestones-children-treated-prior-sma-symptom-onset

title:
novartis.com: Novartis shares zolgensma long term data demonstrating sustained durability 75 years post dosing 100 achievement all assessed milestones children treated prior sma symptom onset


node_name: Zolgensma SMA Presymptomatic Treatment Model
created_at: 2026-04-19 20:11:05
url:
https://medicalresearch.com/one-time-gene-replacement-therapy-with-zolgensma-demonstrates-transformational-benefit-for-spinal-muscular-atrophy-patients

title:
medicalresearch.com: One time gene replacement therapy with zolgensma demonstrates transformational benefit for spinal muscular atrophy patients


title: health.ucdavis.edu
node_name: In Utero Gene Editing Fetal Immune Tolerance
created_at: 2026-04-19 20:11:05
url:
https://health.ucdavis.edu/news/headlines/dream-team-to-develop-in-utero-gene-editing-therapy-for-a-rare-neurological-genetic-disorder/2025/09


title: uniqure.gcs-web.com: Uniqure announces positive topline results pivotal phase iii
node_name: Huntington's Disease Intrastriatal Gene Silencing
created_at: 2026-04-19 20:11:05
url:
https://uniqure.gcs-web.com/news-releases/news-release-details/uniqure-announces-positive-topline-results-pivotal-phase-iii


title: neurologylive.com: Fda reverses course amt 130 citing insufficient external data for submission
node_name: Huntington's Disease Intrastriatal Gene Silencing
created_at: 2026-04-19 20:11:05
url:
https://www.neurologylive.com/view/fda-reverses-course-amt-130-citing-insufficient-external-data-for-submission


title: biospace.com: Fda reversals in rare disease space highlight confusion around external controls
node_name: FDA Sham Surgery RCT Mandate for CNS Gene Therapy
created_at: 2026-04-19 20:11:05
url:
https://www.biospace.com/fda/fda-reversals-in-rare-disease-space-highlight-confusion-around-external-controls


title: investorrelations.sarepta.com: Sarepta announces approval begin endeavor cohort 8 evaluate
node_name: Sirolimus Immunosuppression Protocol for AAV Safety
created_at: 2026-04-19 20:11:05
url:
https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-announces-approval-begin-endeavor-cohort-8-evaluate


node_name: Antibody-Oligonucleotide Conjugate Muscle Delivery Platform
created_at: 2026-04-19 20:11:05
url:
https://www.cgtlive.com/view/patients-dmd-treated-avidity-biosciences-antibody-oligonucleotide-conjugate-del-zota-functional-improvements

title:
cgtlive.com: Patients dmd treated avidity biosciences antibody oligonucleotide conjugate del zota functional improvements


node_name: Antibody-Oligonucleotide Conjugate Muscle Delivery Platform
created_at: 2026-04-19 20:11:05
url:
https://www.neurologylive.com/view/del-zota-produced-statistically-significant-increases-in-exon-skipping-and-dystrophin-levels-in-explore44-a-phase-1-2-study-in-individuals-with-dmd44m

title:
neurologylive.com: Del zota produced statistically significant increases in exon skipping and dystrophin levels in explore44 a phase 1 2 study in individuals with dmd44m


node_name: Antibody-Oligonucleotide Conjugate Muscle Delivery Platform
created_at: 2026-04-19 20:11:05
url:
https://www.prnewswire.com/news-releases/avidity-biosciences-receives-fda-breakthrough-therapy-designation-for-delpacibart-zotadirsen-del-zota-for-the-treatment-of-dmd-in-people-with-mutations-amenable-to-exon-44-skipping-302511334.html

title:
prnewswire.com: Avidity biosciences receives fda breakthrough therapy designation for delpacibart zotadirsen del zota for the treatment of dmd in people with mutations amenable to exon 44 skipping 302511334


node_name: CRISPR-Edited TIL Solid Tumor CISH Knockout
created_at: 2026-04-19 20:11:05
url:
https://www.intimabioscience.com/press_releases/data-from-first-in-human-trial-targeting-cish-a-novel-immune-checkpoint-in-patients-with-metastatic-colorectal-cancer/

title:
intimabioscience.com: Data from first in human trial targeting cish a novel immune checkpoint in patients with metastatic colorectal cancer


node_name: CRISPR-Edited TIL Solid Tumor CISH Knockout
created_at: 2026-04-19 20:11:05
url:
https://ascopost.com/news/may-2025/crispr-cas9-edited-tils-targeting-intracellular-immune-checkpoint-cish-in-metastatic-colorectal-cancer/

title:
ascopost.com: Crispr cas9 edited tils targeting intracellular immune checkpoint cish in metastatic colorectal cancer


title: ir.crisprtx.com: Crispr therapeutics presents new preclinical data ctx460tm
node_name: AATD Multi-Modality Battleground
created_at: 2026-04-19 20:11:05
url:
https://ir.crisprtx.com/news-releases/news-release-details/crispr-therapeutics-presents-new-preclinical-data-ctx460tm/


title: investors.primemedicine.com: Prime medicine unveils program treatment alpha 1 antitrypsin
node_name: AATD Multi-Modality Battleground
created_at: 2026-04-19 20:11:05
url:
https://investors.primemedicine.com/news-releases/news-release-details/prime-medicine-unveils-program-treatment-alpha-1-antitrypsin


node_name: GLP-1 x CRISPR Cardiometabolic Convergence
created_at: 2026-04-19 20:11:05
url:
https://www.geneonline.com/the-2026-cardiometabolic-and-weight-loss-biotech-watchlist-trials-that-could-mint-the-next-trillion-dollar-giant/

title:
geneonline.com: The 2026 cardiometabolic and weight loss biotech watchlist trials that could mint the next trillion dollar giant


node_name: CRISPR IP Wars: Broad vs CVC Patent Control
created_at: 2026-04-19 20:11:05
url:
https://news.berkeley.edu/2026/03/26/ptab-sides-with-broad-institute-over-university-of-california-on-patent-priority-for-use-of-crispr-in-eukaryotic-cells/

title:
news.berkeley.edu: Ptab sides with broad institute over university of california on patent priority for use of crispr in eukaryotic cells


node_name: CRISPR IP Wars: Broad vs CVC Patent Control
created_at: 2026-04-19 20:11:05
url:
https://www.biospace.com/business/ongoing-crispr-patent-dispute-complicates-licensing-but-hasnt-deterred-gene-editing-investment

title:
biospace.com: Ongoing crispr patent dispute complicates licensing but hasnt deterred gene editing investment


node_name: In Vivo HSC Editing Without Myeloablation
created_at: 2026-04-19 20:11:05
url:
https://crisprmedicinenews.com/news/editing-stem-cells-in-vivo-a-major-stride-in-gene-therapy-for-blood-disorders/

title:
crisprmedicinenews.com: Editing stem cells in vivo a major stride in gene therapy for blood disorders


node_name: Lentiviral Insertional Mutagenesis Cancer Risk
created_at: 2026-04-19 20:11:05
url:
https://www.pharmaceutical-technology.com/news/bluebird-bios-skysona-led-to-seven-cases-of-blood-cancer-in-gene-therapy-trials/

title:
pharmaceutical-technology.com: Bluebird bios skysona led to seven cases of blood cancer in gene therapy trials


title: investor.bluebirdbio.com: Bluebird bio provides updated findings reported case acute
node_name: Lentiviral Insertional Mutagenesis Cancer Risk
created_at: 2026-04-19 20:11:05
url:
https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-provides-updated-findings-reported-case-acute


node_name: RNAi vs CRISPR Liver Disease Head-to-Head
created_at: 2026-04-19 20:11:05
url:
https://markets.financialcontent.com/stocks/article/finterra-2026-2-12-alnylam-pharmaceuticals-alny-the-rnai-pioneers-2026-breakout-and-the-battle-for-cardiovascular-dominance

title:
markets.financialcontent.com: Finterra 2026 2 12 alnylam pharmaceuticals alny the rnai pioneers 2026 breakout and the battle for cardiovascular dominance


title: simplywall.st: Did new vutrisiran data and fcf breakeven just shift alnylam
node_name: RNAi vs CRISPR Liver Disease Head-to-Head
created_at: 2026-04-19 20:11:05
url:
https://simplywall.st/stocks/us/pharmaceuticals-biotech/nasdaq-alny/alnylam-pharmaceuticals/news/did-new-vutrisiran-data-and-fcf-breakeven-just-shift-alnylam


title: ir.ionis.com: Fda approves qalsodytm tofersen first treatment targeting
node_name: ALS Genetic Subtype Fragmentation Problem
created_at: 2026-04-19 20:11:05
url:
https://ir.ionis.com/news-releases/news-release-details/fda-approves-qalsodytm-tofersen-first-treatment-targeting


title: investors.biogen.com: Journal american medical association jama neurology publishes
node_name: ALS Genetic Subtype Fragmentation Problem
created_at: 2026-04-19 20:11:05
url:
https://investors.biogen.com/news-releases/news-release-details/journal-american-medical-association-jama-neurology-publishes


node_name: Orphan Drug Act Pricing Architecture
created_at: 2026-04-19 20:11:05
url:
https://www.fiercehealthcare.com/regulatory/expanded-price-negotiation-exemption-orphan-drugs-cost-medicare-88b-over-10-years-cbo

title:
fiercehealthcare.com: Expanded price negotiation exemption orphan drugs cost medicare 88b over 10 years cbo


title: morganlewis.com: Orphan drugs big breaks the quiet carve out in the one big beautiful bill act
node_name: Orphan Drug Act Pricing Architecture
created_at: 2026-04-19 20:11:05
url:
https://www.morganlewis.com/blogs/asprescribed/2025/07/orphan-drugs-big-breaks-the-quiet-carve-out-in-the-one-big-beautiful-bill-act


title: investor.lilly.com: Lilly acquire verve therapeutics advance one time treatments
node_name: Lilly Cardiometabolic Prevention Stack
created_at: 2026-04-19 20:11:05
url:
https://investor.lilly.com/news-releases/news-release-details/lilly-acquire-verve-therapeutics-advance-one-time-treatments


node_name: Lilly Cardiometabolic Prevention Stack
created_at: 2026-04-19 20:11:05
url:
https://crisprmedicinenews.com/news/eli-lilly-to-acquire-verve-therapeutics-to-advance-one-time-cardiovascular-treatments/

title:
crisprmedicinenews.com: Eli lilly to acquire verve therapeutics to advance one time cardiovascular treatments


node_name: Epigenetic Editing (EPI-321 FSHD): Silencing Without Cutting
created_at: 2026-04-19 20:11:05
url:
https://epicrispr.com/epicrispr-biotechnologies-doses-first-patient-in-first-in-human-clinical-trial-of-epi-321-for-facioscapulohumeral-muscular-dystrophy/

title:
epicrispr.com: Epicrispr biotechnologies doses first patient in first in human clinical trial of epi 321 for facioscapulohumeral muscular dystrophy


node_name: NIH/DOGE Research Funding Disruption on Gene Therapy Pipeline
created_at: 2026-04-19 20:11:05
url:
https://thebulletin.org/premium/2025-05/the-impact-of-doges-funding-cuts-on-biomedical-research-from-the-point-of-view-of-former-nih-director-monica-bertagnolli/

title:
thebulletin.org: The impact of doges funding cuts on biomedical research from the point of view of former nih director monica bertagnolli


title: infectiousdiseaseadvisor.com: Nih research funding drops 1b under trump administration
node_name: NIH/DOGE Research Funding Disruption on Gene Therapy Pipeline
created_at: 2026-04-19 20:11:05
url:
https://www.infectiousdiseaseadvisor.com/news/nih-research-funding-drops-1b-under-trump-administration/


node_name: AAV Systemic Hepatotoxicity Death Mechanism
created_at: 2026-04-19 20:11:05
url:
https://www.fda.gov/vaccines-blood-biologics/safety-availability-biologics/fda-investigating-deaths-due-acute-liver-failure-following-treatment-sareptas-aavrh74-gene-therapies

title:
fda.gov: Fda investigating deaths due acute liver failure following treatment sareptas aavrh74 gene therapies


title: neurologylive.com: Third patient death leads significant concerns sarepta gene therapy program
node_name: AAV Systemic Hepatotoxicity Death Mechanism
created_at: 2026-04-19 20:11:05
url:
https://www.neurologylive.com/view/third-patient-death-leads-significant-concerns-sarepta-gene-therapy-program


title: cgtlive.com: Patient dies acute liver failure treatment sarepta dmd gene therapy elevidys
node_name: AAV Systemic Hepatotoxicity Death Mechanism
created_at: 2026-04-19 20:11:05
url:
https://www.cgtlive.com/view/patient-dies-acute-liver-failure-treatment-sarepta-dmd-gene-therapy-elevidys


node_name: AI-Designed CRISPR: OpenCRISPR Protein Language Model
created_at: 2026-04-19 20:11:05
url:
https://www.reprocell.com/blog/opencrispr-1-the-first-ai-designed-crispr-editor-for-high-precision-gene-cell-therapy

title:
reprocell.com: Opencrispr 1 the first ai designed crispr editor for high precision gene cell therapy


node_name: LNP Ionizable Lipid IP Concentration: Acuitas Chokepoint
created_at: 2026-04-19 20:11:05
url:
https://www.bayer.com/media/en-us/bayer-strengthens-gene-therapy-portfolio-with-lipid-nanoparticle-technology-from-acuitas-therapeutics/

title:
bayer.com: Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from acuitas therapeutics


title: pfizer.com: Pfizer enters agreement acuitas therapeutics lipid
node_name: LNP Ionizable Lipid IP Concentration: Acuitas Chokepoint
created_at: 2026-04-19 20:11:05
url:
https://www.pfizer.com/news/press-release/press-release-detail/pfizer-enters-agreement-acuitas-therapeutics-lipid


title: carlyle.com: Bluebird bio announces completion acquisition carlyle and sk
node_name: Gene Therapy Biotech Capital Destruction and PE Extraction Cycle
created_at: 2026-04-19 20:11:05
url:
https://www.carlyle.com/media-room/news-release-archive/bluebird-bio-announces-completion-acquisition-carlyle-and-sk


node_name: China CRISPR Clinical Data Engine: Speed vs Safety Asymmetry
created_at: 2026-04-19 20:11:05
url:
https://www.geneticsandsociety.org/article/after-crispr-baby-scandal-shut-down-work-years-china-gene-editing-companies-are-restarting

title:
geneticsandsociety.org: After crispr baby scandal shut down work years china gene editing companies are restarting


title: inbt.jhu.edu: Machine learning unlocks next generation lipid nanoparticles for safer gene editing
node_name: AI-Guided LNP and CRISPR Design Acceleration
created_at: 2026-04-19 20:11:05
url:
https://inbt.jhu.edu/machine-learning-unlocks-next-generation-lipid-nanoparticles-for-safer-gene-editing/


node_name: CMS CGT Access Model Outcomes-Based Payment
created_at: 2026-04-19 20:11:05
url:
https://www.certara.com/blog/cms-cell-and-gene-therapy-cgt-access-model-explained-the-most-significant-drug-pricing-legislation-youve-never-heard-of/

title:
certara.com: Cms cell and gene therapy cgt access model explained the most significant drug pricing legislation youve never heard of


title: globenewswire.com: Gene Therapy Scaling Up for Commercial Success with CDMO Collaborations
node_name: China Gene Therapy Manufacturing Cost Wedge
created_at: 2026-04-19 20:11:05
url:
https://www.globenewswire.com/news-release/2026/02/11/3236333/0/en/Gene-Therapy-Scaling-Up-for-Commercial-Success-with-CDMO-Collaborations.html


title: biospace.com: Viral vectors at the vanguard inside the future factory of gene therapy
node_name: China Gene Therapy Manufacturing Cost Wedge
created_at: 2026-04-19 20:11:05
url:
https://www.biospace.com/press-releases/viral-vectors-at-the-vanguard-inside-the-future-factory-of-gene-therapy


title: agcbio.com: Trends shaping the future of cell and gene therapy manufacturing
node_name: China Gene Therapy Manufacturing Cost Wedge
created_at: 2026-04-19 20:11:05
url:
https://www.agcbio.com/biopharma-blog/trends-shaping-the-future-of-cell-and-gene-therapy-manufacturing


title: insights.bio: Addressing challenges in aav manufacturing scaleup for costeffective gene therapies
node_name: AAV vs LNP Manufacturing Learning Curve Divergence
created_at: 2026-04-19 20:11:05
url:
https://www.insights.bio/cell-and-gene-therapy-insights/journal/article/3445/addressing-challenges-in-aav-manufacturing-scaleup-for-costeffective-gene-therapies


node_name: CRISPR Xenotransplantation: 69-Edit Pig Organ Platform
created_at: 2026-04-19 20:11:05
url:
https://egenesisbio.com/news-media/press-releases/egenesis-announces-ind-clearance-for-eigen-2784-in-kidney-transplant-and-landmark-patient-updates-in-ongoing-expanded-access-study

title:
egenesisbio.com: Egenesis announces ind clearance for eigen 2784 in kidney transplant and landmark patient updates in ongoing expanded access study


node_name: CRISPR Xenotransplantation: 69-Edit Pig Organ Platform
created_at: 2026-04-19 20:11:05
url:
https://crisprmedicinenews.com/news/egenesis-receives-fda-clearance-for-gene-edited-pig-kidney-trial-in-end-stage-kidney-disease-patient/

title:
crisprmedicinenews.com: Egenesis receives fda clearance for gene edited pig kidney trial in end stage kidney disease patient


title: biospace.com: Rna editing hits the clinic fueling new hope for rare and common diseases
node_name: ADAR RNA Editing: Reversible Transcriptome Medicine
created_at: 2026-04-19 20:11:05
url:
https://www.biospace.com/drug-development/rna-editing-hits-the-clinic-fueling-new-hope-for-rare-and-common-diseases


node_name: AAV Seroprevalence: The Hidden Patient Exclusion Cap
created_at: 2026-04-19 20:11:05
url:
https://www.asokanlab.org/post/complete-neutralizing-antibody-evasion-by-serodivergent-non-mammalian-aavs-enables-gene-therapy-redosing

title:
asokanlab.org: Complete neutralizing antibody evasion by serodivergent non mammalian aavs enables gene therapy redosing


title: parexel.com: Gene therapy are high costs and manufacturing complexities impeding progress
node_name: Gene Therapy FOAK-NOAK Manufacturing Cost Cliff
created_at: 2026-04-19 20:11:05
url:
https://www.parexel.com/insights/blog/gene-therapy-are-high-costs-and-manufacturing-complexities-impeding-progress


title: cgtlive.com: Verve therapeutics base editing therapy verve 102 reduces ldl c patients hefh cad
node_name: PCSK9 Base Editing Chronic Medication Disruption
created_at: 2026-04-19 20:11:05
url:
https://www.cgtlive.com/view/verve-therapeutics-base-editing-therapy-verve-102-reduces-ldl-c-patients-hefh-cad


node_name: India BIRSA 101 Affordable CRISPR Model
created_at: 2026-04-19 20:11:05
url:
https://www.biospectrumindia.com/news/16/26952/india-launches-first-indigenous-crispr-based-gene-therapy-for-sickle-cell-disease.html

title:
biospectrumindia.com: India launches first indigenous crispr based gene therapy for sickle cell disease


title: phacilitate.com: Advanced therapies week 2025 developing advanced therapies saudi arabia
node_name: Middle East Sovereign Wealth Gene Therapy Diversification
created_at: 2026-04-19 20:11:05
url:
https://www.phacilitate.com/insights-resources/advanced-therapies-week-2025-developing-advanced-therapies-saudi-arabia


title: pharmaboardroom.com: Middle eastern sovereign wealth funds continue pharma investment push
node_name: Middle East Sovereign Wealth Gene Therapy Diversification
created_at: 2026-04-19 20:11:05
url:
https://pharmaboardroom.com/articles/middle-eastern-sovereign-wealth-funds-continue-pharma-investment-push/


title: biospace.com: Lilly validates gene editing space with 1 3b verve buy but analysts are skeptical
node_name: VERVE-102 Cardiovascular Base Editing Mass Market Shift
created_at: 2026-04-19 20:11:05
url:
https://www.biospace.com/business/lilly-validates-gene-editing-space-with-1-3b-verve-buy-but-analysts-are-skeptical


node_name: AI-Designed AAV Capsid Engineering
created_at: 2026-04-19 20:11:05
url:
https://www.businesswire.com/news/home/20260408079855/en/Dyno-Therapeutics-Announces-Capsid-License-Exercised-by-Astellas-for-Skeletal-Muscle-Targeted-Gene-Delivery-Validating-AI-Powered-Technology-for-Biological-Sequence-Design

title:
businesswire.com: Dyno Therapeutics Announces Capsid License Exercised by Astellas for Skeletal Muscle Targeted Gene Delivery Validating AI Powered Technology for Biological Sequence Design


node_name: AI-Designed AAV Capsid Engineering
created_at: 2026-04-19 20:11:05
url:
https://www.biospace.com/press-releases/dyno-therapeutics-launches-three-breakthrough-capsid-delivery-vectors-for-next-generation-eye-muscle-and-cns-gene-therapies-at-the-2025-american-society-of-gene-cell-therapy-asgct-annual-meeting

title:
biospace.com: Dyno therapeutics launches three breakthrough capsid delivery vectors for next generation eye muscle and cns gene therapies at the 2025 american society of gene cell therapy asgct annual meeting


title: mercer.com: Glp 1 considerations for 2026 your questions answered
node_name: GLP-1 vs Gene Therapy Payer Budget Zero-Sum
created_at: 2026-04-19 20:11:05
url:
https://www.mercer.com/en-us/insights/us-health-news/glp-1-considerations-for-2026-your-questions-answered/


title: ajmc.com: With approval of car tcell therapy comes the next challenge payer coverage
node_name: CAR-T Cancer Payer Reimbursement Template
created_at: 2026-04-19 20:11:05
url:
https://www.ajmc.com/view/with-approval-of-car-tcell-therapy-comes-the-next-challenge-payer-coverage


node_name: CRISPR Cardiovascular In Vivo Editing
created_at: 2026-04-19 20:11:05
url:
https://www.globenewswire.com/news-release/2025/11/08/3184062/0/en/CRISPR-Therapeutics-Announces-Positive-Phase-1-Clinical-Data-for-CTX310-Demonstrating-Deep-and-Durable-ANGPTL3-Editing-Triglyceride-and-Lipid-Lowering.html

title:
globenewswire.com: CRISPR Therapeutics Announces Positive Phase 1 Clinical Data for CTX310 Demonstrating Deep and Durable ANGPTL3 Editing Triglyceride and Lipid Lowering


title: cgtlive.com: Wave life sciences achieves rna editing trial wve 006 alpha 1 antitrypsin deficiency
node_name: ADAR RNA Editing Platform
created_at: 2026-04-19 20:11:05
url:
https://www.cgtlive.com/view/wave-life-sciences-achieves-rna-editing-trial-wve-006-alpha-1-antitrypsin-deficiency


node_name: ADAR RNA Editing Platform
created_at: 2026-04-19 20:11:05
url:
https://www.globenewswire.com/news-release/2026/02/02/3230159/0/en/Wave-Life-Sciences-Announces-Plans-to-Accelerate-Regulatory-Engagement-with-Full-Control-of-WVE-006-for-Alpha-1-Antitrypsin-Deficiency.html

title:
globenewswire.com: Wave Life Sciences Announces Plans to Accelerate Regulatory Engagement with Full Control of WVE 006 for Alpha 1 Antitrypsin Deficiency


title: advi.com: 2026 life sciences forecast ai next gen cell gene therapy and policy disruption
node_name: Gene Therapy Subscription Destroyer Pattern
created_at: 2026-04-19 20:11:05
url:
https://www.advi.com/insight/2026-life-sciences-forecast-ai-next-gen-cell-gene-therapy-and-policy-disruption/


title: ir.wavelifesciences.com: Wave life sciences announces plans accelerate regulatory
node_name: GalNAc Targeted Oligonucleotide Liver Delivery
created_at: 2026-04-19 20:11:05
url:
https://ir.wavelifesciences.com/news-releases/news-release-details/wave-life-sciences-announces-plans-accelerate-regulatory


title: fda.gov: Fda approves gene therapy treatment spinal muscular atrophy
node_name: Intrathecal CNS AAV Delivery
created_at: 2026-04-19 20:11:05
url:
https://www.fda.gov/news-events/press-announcements/fda-approves-gene-therapy-treatment-spinal-muscular-atrophy


title: practicalneurology.com: 2484610
node_name: Intrathecal CNS AAV Delivery
created_at: 2026-04-19 20:11:05
url:
https://practicalneurology.com/news/intrathecal-gene-therapy-approved-for-adults-older-children-with-spinal-muscular-atrophy/2484610/


title: bioprocessintl.com: Overcoming aav manufacturing challenges movement toward plug and play solutions
node_name: AAV Empty Capsid Manufacturing Barrier
created_at: 2026-04-19 20:11:05
url:
https://www.bioprocessintl.com/cell-therapies/overcoming-aav-manufacturing-challenges-movement-toward-plug-and-play-solutions


node_name: Neurodegenerative Disease Gene Therapy Frontier
created_at: 2026-04-19 20:11:05
url:
https://xtalks.com/top-clinical-trials-to-watch-in-2026-whats-coming-in-alzheimers-als-and-gene-editing-4531/

title:
xtalks.com: Top clinical trials to watch in 2026 whats coming in alzheimers als and gene editing 4531


title: ir.voyagertherapeutics.com: Voyager presents robust preclinical data tau targeting gene
node_name: Neurodegenerative Disease Gene Therapy Frontier
created_at: 2026-04-19 20:11:05
url:
https://ir.voyagertherapeutics.com/news-releases/news-release-details/voyager-presents-robust-preclinical-data-tau-targeting-gene


title: pharmasalmanac.com: Gene therapy at a crossroads rethinking aav amid industry resets
node_name: Gene Therapy Sector VC Winter 2024-2026
created_at: 2026-04-19 20:11:05
url:
https://www.pharmasalmanac.com/articles/gene-therapy-at-a-crossroads-rethinking-aav-amid-industry-resets


title: fiercebiotech.com: Q3 biopharma layoffs hold steady setting 2025 break last years high
node_name: Gene Therapy Sector VC Winter 2024-2026
created_at: 2026-04-19 20:11:05
url:
https://www.fiercebiotech.com/biotech/q3-biopharma-layoffs-hold-steady-setting-2025-break-last-years-high


node_name: AAV Intracranial Oncogenesis Safety Signal
created_at: 2026-04-19 20:11:05
url:
https://www.neurologylive.com/view/fda-approves-new-intrathecal-administration-route-spinal-muscular-atrophy-gene-therapy

title:
neurologylive.com: Fda approves new intrathecal administration route spinal muscular atrophy gene therapy


node_name: Casgevy HSC Mobilization Bottleneck
created_at: 2026-04-19 20:11:05
url:
https://ashpublications.org/bloodadvances/article/9/24/6524/546481/Challenges-and-limitations-of-mobilization-and-stem-cell-collection-for-gene-therapy-of-sickle-cell-disease

title:
ashpublications.org: Challenges and limitations of mobilization and stem cell collection for gene therapy of sickle cell disease


title: businesswire.com: Scribe Therapeutics Projected to Enter the Clinic in Mid 2026 with STX 1150
node_name: Epigenome Editing Durable Gene Silencing
created_at: 2026-04-19 20:11:05
url:
https://www.businesswire.com/news/home/20260120569064/en/Scribe-Therapeutics-Projected-to-Enter-the-Clinic-in-Mid-2026-with-STX-1150


node_name: Epigenome Editing Durable Gene Silencing
created_at: 2026-04-19 20:11:05
url:
https://crisprmedicinenews.com/press-release-service/card/scribe-therapeutics-projected-to-enter-the-clinic-in-mid-2026-with-stx-1150-a-pcsk9-targeting-crispr-epigenetic-silencing-therapy-for-durable-ldl-c-reduction/

title:
crisprmedicinenews.com: Scribe therapeutics projected to enter the clinic in mid 2026 with stx 1150 a pcsk9 targeting crispr epigenetic silencing therapy for durable ldl c reduction


title: hklaw.com: Fda publishes new draft guidance on regenerative medicine therapies
node_name: RMAT Designation Gene Therapy Regulatory Accelerator
created_at: 2026-04-19 20:11:05
url:
https://www.hklaw.com/en/insights/publications/2025/10/fda-publishes-new-draft-guidance-on-regenerative-medicine-therapies


title: fda.gov: Regenerative medicine advanced therapy designation
node_name: RMAT Designation Gene Therapy Regulatory Accelerator
created_at: 2026-04-19 20:11:05
url:
https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/regenerative-medicine-advanced-therapy-designation


