Gene Editing & Gene Therapy Clinical Trials

Life Biosciences

First-ever human trial of partial epigenetic reprogramming. AAV-delivered OSK (Oct4, Sox2, Klf4) Yamanaka factors administered via intravitreal injection with doxycycline-inducible expression to restore youthful DNA methylation patterns in retinal ganglion cells. Based on Sinclair lab's 2020 Nature paper.

Life Biosciences

Dual-indication arm of the first-ever human epigenetic reprogramming trial. Same ER-100 AAV gene therapy targeting retinal ganglion cell rejuvenation via OSK partial reprogramming. NAION causes sudden optic nerve damage with no approved treatment.

Prime Medicine

In vivo prime editing therapy targeting the most common Wilson's disease mutation (H1069Q in ATP7B). Delivered via LNP to liver. Preclinical data showed normalization of hepatic copper levels by PET imaging.

Prime Medicine

In vivo prime editing to correct the PiZ mutation (E342K in SERPINA1) responsible for AATD. Achieved high editing efficiency and restoration of corrected M-AAT protein to healthy range in humanized mouse models.

Belief BioMed / Takeda China

China's first approved hemophilia B gene therapy, approved by NMPA in April 2025. AAV-based one-time treatment that restored FIX activity and dramatically reduced bleeding episodes and infusion requirements.

CorrectSequence Therapeutics

World's first gene-editing therapy targeting APOC3 for hyperlipidemia. First patient dosed November 2025 in IIT. Patient with chylomicronemia and fasting TG >12.5 mmol/L saw significant triglyceride drop within 3 days after single low-dose administration, with no adverse events.

IGIB / AIIMS Delhi

India's first CRISPR therapy clinical trial, using enFnCas9 (enhanced FnCas9) developed at IGIB. Plans to recruit 3-5 patients from Chhattisgarh for Phase 1. Aims to provide affordable CRISPR therapy for India's large sickle cell population. All preclinical and animal model data completed.

Broad Institute (David Liu lab)

Disease-agnostic prime editing platform that installs optimized suppressor tRNAs to read through premature stop codons. A single composition could treat ~24% of all genetic diseases. Restored 20-70% enzyme activity in Batten, Tay-Sachs, and Niemann-Pick cell models.

CRISPR Therapeutics

CRISPR-edited allogeneic anti-CD19 CAR-T for autoimmune diseases. Phase 1 basket trial in SLE, systemic sclerosis, and inflammatory myositis. First SLE patient achieved drug-free DORIS remission at Month 6.

UC San Francisco

Breakthrough: first method to precisely integrate large DNA sequences at specific sites in human T cells inside the body. Single injection cleared all detectable cancer in nearly all mice within two weeks, with CAR-T cells comprising 40% of immune cells.

Academic (Published NEJM)

First in vivo CD19 CAR-T cell therapy for refractory SLE — engineering immune cells directly inside the body without ex vivo manufacturing. Published in NEJM.

Orchard Therapeutics

Lentiviral gene therapy delivering a functional ARSA gene to patient's own stem cells for MLD, a fatal lysosomal storage disorder. Treats pre-symptomatic late-infantile and early-juvenile forms. Approved in EU as Libmeldy in 2020, US as Lenmeldy in 2024.

HuidaGene Therapeutics

M.U.S.C.L.E. clinical trial for DMD using novel hfCas12Max DNA-editing system. First patient dosed late 2024. HuidaGene is a leading Chinese gene editing company backed by CRISPR pioneer Feng Zhang. Late-breaking data presented at World Muscle Society 2024.

CARsgen Therapeutics

Fully human BCMA-targeting autologous CAR-T approved by NMPA on February 23, 2024 for adult patients with r/r multiple myeloma after 3+ lines of therapy. CARsgen's lead product demonstrating durable responses.

Legend Biotech / GenScript

BCMA-targeting CAR-T therapy approved by NMPA in August 2024 (Approval Number S20240038). Same product as Carvykti approved in the US, but approved separately in China by Legend Biotech, a GenScript subsidiary originally from Nanjing.

CorrectSequence Therapeutics

IND approved by NMPA April 2024. Successfully treated first overseas patient with TDT, achieving sustained transfusion-free status with hemoglobin >120 g/L. Also treated first sickle cell patient with promising results. Dozens of patients treated across indications.

GenAssist Ltd

First DMD patient dosed with base editing drug September 2024 at Peking Union Medical College Hospital. Uses RNA editing-free Targeted AID-mediated Mutagenesis (TAM) cytosine base editor for one-shot systemic administration to permanently restore dystrophin expression. FDA IND clearance received March 2025.

HuidaGene Therapeutics

HERO clinical trial — first-in-human CRISPR RNA-editing therapy for MECP2 duplication syndrome. Open-label multi-dose study at Peking University First Hospital in male patients ages 2-18. First patient dosed June 2024. Received FDA Orphan Drug Designation and EMA Orphan Designation.

Xijing Hospital / Anhui Medical University

Gene-edited pig liver transplants into human recipients. One patient at Anhui Medical University lived 171 days post-transplant (38 days with pig liver), the longest survival with a pig organ. Published in Nature. No hyperacute rejection observed.

eGenesis

CRISPR-edited pig kidneys with 69 genomic edits — removing pig retroviruses, knocking out immunogenic pig genes, and adding human complement regulatory proteins. First FDA-authorized xenotransplantation clinical trial. Pig kidneys transplanted into living human patients.

Vertex Pharmaceuticals

Stem cell-derived, fully differentiated insulin-producing islet cells in an implantable device for Type 1 diabetes. Encapsulated cells produce insulin without immunosuppression. Based on Doug Melton's Harvard stem cell work. VX-880 predecessor showed patients achieving insulin independence.

Vertex / CRISPR Therapeutics

Casgevy approved in Saudi Arabia, Bahrain, and UAE — marking the first CRISPR therapy approvals in the Middle East. Critical for the region which has high prevalence of sickle cell disease and beta-thalassemia.

Prime Medicine / Broad Institute / CF Foundation

Prime editing correction of CFTR F508del (85% of CF patients). Six-optimization approach achieved 58% correction in bronchial epithelial cells, restoring function to Trikafta-level. $39M funding from CF Foundation.

Tune Therapeutics

Epigenetic editing therapy to silence HBV cccDNA without cutting DNA. Uses dead Cas9 fused to epigenetic effectors. Clinical trial approved in New Zealand (November 2024). First epigenetic editing therapy in clinical trials.

HuidaGene

RNA editing therapy for MECP2 duplication syndrome. First patient dosed December 2024. Uses ADAR-based RNA editing approach to correct neurological gene expression without permanent DNA changes.

Wave Life Sciences

Oligonucleotide-based RNA editing that recruits endogenous ADAR enzyme to correct the AATD PiZ mutation at the RNA level. No exogenous protein needed — just a synthetic oligonucleotide. Potentially reversible and re-dosable.

CRISPR Therapeutics / Vertex Pharmaceuticals

First CRISPR-based gene therapy ever approved. Edits BCL11A gene in patient's own stem cells to reactivate fetal hemoglobin production, reducing sickling crises and eliminating transfusion dependence.

bluebird bio

Lentiviral gene therapy that adds a modified beta-globin gene (betaA-T87Q) to patient's own stem cells to produce anti-sickling hemoglobin HbAT87Q. Carries a boxed warning for hematologic malignancy risk.

Sarepta Therapeutics

AAVrh74-based gene therapy delivering a micro-dystrophin transgene for DMD. Received accelerated approval in 2023 for ambulatory patients aged 4-5, then expanded in 2024 to all ambulatory DMD patients regardless of age.

ImmunoACT / IIT Bombay

India's first indigenously developed CAR-T cell therapy, approved by CDSCO in 2023. Uses a humanized anti-CD19 CAR with 4-1BB costimulatory domain. Priced at ~$40,000, dramatically lower than Western CAR-T products ($350,000-$500,000).

Prime Medicine

First prime editing therapy approaching clinical development. Uses prime editors to correct mutations in the CYBB gene that cause X-linked chronic granulomatous disease. Prime editing enables precise insertion/deletion without double-strand breaks.

Krystal Biotech

First FDA-approved topical gene therapy. Uses a non-replicating HSV-1 viral vector to deliver functional COL7A1 gene directly to skin wounds in patients with dystrophic epidermolysis bullosa.

Shanghai YaKe Biotechnology / Chinese hospitals

Dual-targeting BCMA-CD19 compound CAR-T cell therapy for refractory SLE. Published in Nature Medicine. All patients achieved B cell depletion within 1-10 days. 12/13 met Lupus Low Disease Activity State with negative autoantibodies at 3-6 months.

Nanjing Bioheng Biotech

CD7-targeted universal (off-the-shelf) CAR-T using proprietary Cas-V2 system. Genetically modified to avoid fratricide, GvHD, and host rejection. FDA IND cleared March 2025. TENACITY-01 global Phase 1b/2 trial. First off-the-shelf CAR-T for T-cell malignancies.

Heartseed Inc.

First clinical trial of iPSC-derived cardiomyocyte spheroids transplanted directly into the heart. LAPiS trial at Keio University Hospital. iPSCs are differentiated into beating heart muscle cells, formed into spheroids, and injected into damaged heart tissue to regenerate function.

BlueRock Therapeutics (Bayer)

iPSC-derived dopaminergic neurons implanted into the brains of Parkinson's patients to replace the neurons lost in the disease. Phase 1 showed the cells survived, engrafted, and produced dopamine. Phase 2 randomized, controlled trial underway. Acquired by Bayer.

Ultragenyx

AAV8 vector gene therapy expressing human G6PC gene for GSD1a. Rolling BLA completed, supported by 96-week Phase 3 GlucoGene trial data. PDUFA date: March 28, 2026.

Johnson & Johnson / Legend Biotech

BCMA-targeted CAR-T with a unique dual-epitope binding domain (two BCMA-targeting single-domain antibodies) for enhanced avidity. Showed superior results to Abecma in cross-trial comparisons.

CSL Behring / uniQure

AAV5-based gene therapy delivering a Padua variant Factor IX gene for a single-dose treatment of hemophilia B. At $3.5M, became the world's most expensive drug. Provides sustained Factor IX activity levels.

Intellia Therapeutics

In vivo CRISPR therapy targeting the KLKB1 gene in the liver to reduce plasma kallikrein and prevent HAE attacks. Delivered via lipid nanoparticles as a one-time IV infusion.

Beam Therapeutics

First base editing therapy to enter clinical trials. Uses adenine base editing to make a precise A-to-G change in the HBG1/2 promoter region to reactivate fetal hemoglobin production, potentially a more precise alternative to Casgevy's CRISPR approach.

Verve Therapeutics

In vivo base editing therapy to permanently inactivate the PCSK9 gene in the liver, reducing LDL cholesterol. Uses adenine base editing delivered via lipid nanoparticles. Could replace lifelong statin therapy with a single treatment.

Excision BioTherapeutics

First CRISPR therapy aimed at curing HIV. Uses AAV9 to deliver multiplex CRISPR (two guide RNAs) to excise integrated HIV-1 proviral DNA from the host genome. Targets LTR regions of the provirus.

Cure Rare Disease

Personalized CRISPR gene editing therapy designed for a single patient, Terry Horgan, with a specific DMD exon 45 deletion. Used AAV to deliver CRISPR components to skip exon 44. Terry Horgan tragically died days after treatment in October 2022.

Altos Labs

Funded with $3 billion from investors including Jeff Bezos and Yuri Milner. Pursuing partial epigenetic reprogramming using transient expression of Yamanaka factors (Oct4, Sox2, Klf4, c-Myc) to reverse cellular aging without dedifferentiation. Recruited top scientists including Shinya Yamanaka, Juan Carlos Izpisua Belmonte, and Steve Horvath.

Graphite Bio (now Lenz Therapeutics)

CRISPR-based gene correction (not just editing) approach for SCD, using homology-directed repair to directly correct the sickle mutation in the HBB gene to restore normal hemoglobin A production. First patient experienced prolonged pancytopenia.

Curocell

Korea's first domestically developed CAR-T therapy. Phase 2 trial completed for r/r DLBCL. Expected to become first approved CAR-T in Korea. Selected for Korea's accelerated approval pilot project.

Great Ormond Street Hospital / UCL

Base-edited allogeneic CAR-T targeting CD7 for T-ALL. Uses quadruple base editing to create universal donor T cells. 82% of patients achieved deep remission. Published in NEJM.

Bristol Myers Squibb / 2seventy bio

First CAR-T therapy approved for multiple myeloma. Targets B-cell maturation antigen (BCMA) on myeloma cells. Patient T cells are transduced with a lentiviral vector encoding an anti-BCMA CAR.

BlueRock Therapeutics / Bayer

iPSC-derived dopaminergic neuron cell therapy for Parkinson's disease. iPSCs are differentiated into A9 midbrain dopaminergic neurons and surgically implanted into the putamen. Represents the frontier of cell replacement therapy for neurodegeneration.

Vertex Pharmaceuticals

Stem cell-derived, fully differentiated islet cell therapy for type 1 diabetes. Uses CRISPR for cell engineering to create insulin-producing cells. Patients receive islet cells via hepatic portal vein infusion with immunosuppression.

Fosun Kite Biotechnology

China's first commercially approved CAR-T therapy. Anti-CD19 CAR-T cell therapy approved by NMPA June 2021 for adult r/r LBCL after 2+ lines of systemic therapy. Licensed from Kite Pharma's Yescarta and manufactured locally.

JW Therapeutics

Second anti-CD19 CAR-T therapy approved in China. NMPA approved for r/r LBCL (2021), then r/r follicular lymphoma (2022, first CAR-T for FL in China), and r/r mantle cell lymphoma. Multiple indications make it China's most broadly approved CAR-T.

EdiGene (博雅辑因)

First CRISPR gene-editing therapy to receive IND approval from China NMPA (January 2021). Ex vivo autologous HSPC therapy edited with CRISPR-Cas9 to reactivate fetal hemoglobin by disrupting BCL11A-erythroid enhancer. Collaboration with Institute of Hematology & Blood Diseases Hospital.

Gracell Biotechnologies

Dual BCMA/CD19 targeting FasTCAR-T with ultra-fast 22-36 hour manufacturing (vs 2-6 weeks industry norm). Phase 1 IIT in newly diagnosed high-risk MM showed 100% ORR and 100% MRD negativity. NMPA cleared Phase 1/2 trial (Q3 2023). FDA cleared Phase 1b/2 trial.

Intellia Therapeutics

First-ever in vivo CRISPR gene editing therapy administered systemically. Uses lipid nanoparticles to deliver CRISPR-Cas9 to the liver to knock out the TTR gene, reducing toxic misfolded transthyretin protein. A watershed moment for gene editing.

Editas Medicine

First in vivo CRISPR gene editing therapy administered directly in the human body. Subretinal injection of AAV5 carrying S. aureus Cas9 and guides to remove an intronic IVS26 mutation in the CEP290 gene. BRILLIANCE trial showed mixed results.

Novartis Gene Therapies

AAV9-based gene therapy delivering a functional copy of the SMN1 gene via a single IV infusion. Targets motor neurons to restore survival motor neuron protein production. At $2.1M, was the world's most expensive drug at launch.

CARsgen Therapeutics

Anti-Claudin18.2 CAR-T therapy for advanced gastric cancer. One of the first CAR-T therapies to show meaningful activity in solid tumors. GCAR-T clinical program spans China, US, and Canada.

University of Pennsylvania (Carl June)

First US clinical trial using CRISPR-edited cells. Used CRISPR to knock out TRAC, TRBC, and PD-1 in patient T cells, then added an NY-ESO-1 cancer-targeting TCR via lentivirus. Triple-edited T cells for enhanced anti-tumor activity.

AnGes / Mitsubishi Tanabe Pharma

Japan's first gene therapy product, granted conditional time-limited approval in September 2019 for ulcers in peripheral arterial disease. Later withdrawn by sponsor due to failure to demonstrate efficacy in post-marketing trial.

Southern University of Science and Technology (unauthorized)

He Jiankui secretly used CRISPR-Cas9 to edit the CCR5 gene in human embryos to confer HIV resistance, resulting in the birth of twins Lulu and Nana (Nov 2018) and a third baby (2019). Widely condemned as reckless and unethical. He was imprisoned for 3 years.

Spark Therapeutics / Roche

First FDA-approved gene therapy for a genetic disease. Delivers a functional RPE65 gene via AAV2 vector directly into the subretinal space to restore vision in patients with biallelic RPE65-mediated inherited retinal dystrophy.

Novartis

First CAR-T cell therapy approved by the FDA. Patient's T cells are engineered with a lentiviral vector to express a chimeric antigen receptor targeting CD19 on B cells. Landmark approval for pediatric/young adult ALL and adult DLBCL.

Gilead Sciences / Kite Pharma

Anti-CD19 CAR-T therapy for relapsed or refractory large B-cell lymphoma. Uses a retroviral vector to engineer patient T cells. Later approved as second-line treatment after ZUMA-7 trial results.

Cellectis / Servier

First allogeneic CAR-T therapy to enter clinical trials. Uses TALEN gene editing to knock out TRAC and CD52 genes in donor T cells, enabling off-the-shelf anti-CD19 CAR-T without graft-versus-host disease.

Astellas (formerly Audentes)

AAV8-based gene therapy delivering MTM1 gene for fatal XLMTM. Trial placed on clinical hold after 4 patient deaths from hepatotoxicity related to high-dose AAV. Raised critical safety questions about AAV dose limits.

Sangamo Therapeutics

First in vivo gene editing trial using zinc finger nucleases. AAV-delivered ZFN pair targets the albumin locus in hepatocytes for site-specific insertion of the IDS gene. Pioneered the concept of in vivo genome editing.

Sun Yat-Sen University First Affiliated Hospital

First clinical trial to use CRISPR to edit cells while inside the human body (in vivo). Targeted HPV E6/E7 viral genes in cervical tissue to treat HPV-related pre-cancerous lesions. NCT03057912.

GSK / Orchard Therapeutics

First ex vivo stem cell gene therapy approved in the EU. Uses a retroviral vector to insert a functional ADA gene into the patient's own bone marrow stem cells. Available only at a single center in Milan.

Sichuan University (Lu You)

First-ever CRISPR-edited cells injected into a human. Used CRISPR-Cas9 to knock out PD-1 gene in patient's T cells before reinfusion. Beat the US to become the first human CRISPR clinical trial, igniting a 'CRISPR race' between the US and China.

BioViva Sciences (Liz Parrish, CEO)

CEO Liz Parrish traveled to Colombia to self-administer two experimental gene therapies: an AAV-delivered telomerase (hTERT) gene therapy and a myostatin inhibitor (follistatin) gene therapy. Highly controversial self-experimentation outside regulatory oversight.

uniQure

First gene therapy approved in the Western world (EU, 2012). AAV1 vector delivering lipoprotein lipase gene. Priced at $1M, it was used on only one commercial patient. Marketing authorization was not renewed in 2017 due to commercial failure.

Sangamo Therapeutics

Pioneering zinc finger nuclease trial to knock out CCR5 in patient's own T cells ex vivo, then reinfuse them. Inspired by the 'Berlin Patient' (Timothy Ray Brown), who was cured of HIV via a CCR5-delta32 bone marrow transplant.

Shanghai Sunway Biotech

World's first approved oncolytic virus therapy, approved by China SFDA in November 2005. Genetically modified adenovirus that selectively replicates in and kills tumor cells with dysfunctional p53 gene. Predecessor to modern oncolytic virus therapies.

Shenzhen SiBiono GeneTech

The world's first commercially approved gene therapy product, approved by China SFDA in October 2003. Uses adenoviral vector to deliver functional p53 tumor suppressor gene. Over 20 years on the market with documented 12+ year clinical follow-up.

University of Pennsylvania (James Wilson)

18-year-old Jesse Gelsinger died 4 days after receiving a high-dose adenoviral vector carrying the OTC gene, due to a massive inflammatory immune response (systemic inflammatory response syndrome). His death led to major reforms in gene therapy oversight.

Necker Hospital / INSERM (Alain Fischer, Marina Cavazzana)

First clear gene therapy cure: 'bubble boy' disease (SCID-X1) patients received retroviral gene therapy inserting the gamma-c cytokine receptor gene into their bone marrow stem cells. While curative, insertional mutagenesis caused leukemia in 5 of 20 patients.

NIH (W. French Anderson, Michael Blaese)

First approved human gene therapy trial in history. Four-year-old Ashanti DeSilva received her own T cells modified with a retroviral vector carrying a functional ADA gene. Marked the birth of gene therapy as a field.