Gene Editing & Gene Therapy Disease Landscape

94.2% overall response rate; 68.3% stringent complete response rate; comparable to Carvykti

71% complete remission rate in relapsed/refractory ALL; comparable efficacy at 1/10th the cost of Western CAR-T

53% complete response rate at 3 months; many responses durable at 12 months; alternative to cystectomy

Comparable efficacy to US Yescarta data; first CAR-T commercial launch in China

73% overall response rate; 53% complete response rate; lower rates of severe CRS compared to other CAR-Ts

81.4% ORR and 67.8% CR in MCL study; first CAR-T approved for follicular lymphoma in China

91% overall response rate; 68% complete response rate

82% overall remission rate in ALL; 50% overall response rate in DLBCL

83% overall response rate; 58% complete response rate; 5-year OS of 42.6%

Enhanced tumor response when combined with chemotherapy; pioneered oncolytic virus field

Demonstrated tumor regression with radiotherapy/chemotherapy; 20+ years of commercial use worldwide

57.1% overall response rate in heavily pretreated gastric cancer; median PFS 3.7 months; landmark for CAR-T in solid tumors

64.1% complete remission rate with 91.7% MRD-negative status in earlier studies

Responses observed in both T-cell lymphoma and renal cell carcinoma; proof-of-concept for CRISPR-edited allogeneic CAR-T in solid tumors

100% ORR, 100% MRD negativity, 87.5% sCR in newly diagnosed MM; ultra-fast manufacturing

68% ORR overall; 83% at recommended Phase 2 dose; 100% in target population

67% overall response rate with manageable safety profile; demonstrated feasibility of allogeneic CRISPR CAR-T

Enhanced efficacy with rapid 72-hour manufacturing process

88% overall response rate; 58% complete response rate at 3 months; durable responses in some patients

82% deep remission rate; gene-edited CAR-T cells erased aggressive T-ALL

All 3 patients showed engraftment of edited cells persisting for 9+ months; no major safety issues; established US clinical precedent for CRISPR

Phase 1 ongoing; first allogeneic CAR-T for myeloid malignancies

~40% tumor response rate in heavily pretreated esophageal cancer patients

Demonstrated feasibility of in vivo CRISPR gene editing in humans

Treatment was safe and feasible; modest clinical benefit; established safety precedent for CRISPR in humans

Single injection cleared cancer in mice; 40% immune cell conversion

Platform technology with multiple programs in IND-enabling studies; $100M+ Sanofi deal for in vivo gene editing

Mean FIX activity 55.08 IU/dL; ABR reduced to 0.6; infusions dropped from 58.2/year to 2.9/year

88% of patients achieved complete resolution of vaso-occlusive events 6-18 months post-infusion

Mean Factor IX activity of 25% at 15 months; 64% reduction in annualized bleed rate

First CRISPR therapy approved in Middle East; 50 active sites globally

97% of SCD patients free from vaso-occlusive crises for 12+ months; 93% of beta-thal patients transfusion-free

89% of patients achieved transfusion independence with sustained hemoglobin levels

Mean Factor VIII activity 42 IU/dL at year 1; declined to ~15 IU/dL by year 3 but still clinically meaningful; 84% reduction in annualized bleed rate

Demonstrated proof of concept for lentiviral SCD gene therapy; complete VOC resolution in 94% of patients in updated protocol group C

Trial initiated in 2024; first patients expected to be treated in 2025

Transfusion independence in TDT patients; hemoglobin stabilized >120 g/L

Beta-thal patient achieved complete transfusion independence for 19+ months with fetal hemoglobin at ~60% of total hemoglobin

Pre-symptomatic late-infantile patients showed near-normal cognitive and motor development; 100% survival at 6+ years vs. ~50% in natural history

Significant micro-dystrophin expression in muscle biopsies; functional improvements in NSAA score at 4 years

90% of patients alive and free of major functional disability at 24 months; comparable outcomes to allogeneic transplant without GVHD risk

91% of patients alive and event-free at 14 months vs. 25% natural history; most achieved motor milestones never seen in untreated SMA1

Single dose reduced serum TTR by up to 93% at 28 days; sustained reductions at 2+ years; Phase 3 MAGNITUDE trial ongoing

Phase 1: iPSC-derived neurons survived 1+ year in patient brains; motor symptom improvement

Robust beta-sarcoglycan expression in muscle biopsies; functional improvements maintained at 4 years

Dramatic improvements in surviving patients (some breathing independently for first time); but 4 deaths from hepatic failure at higher doses prompted hold

Preclinical: precise MECP2 mRNA knockdown in mouse and NHP models; improved phenotypes and prolonged survival

Five different AHC mutations corrected in mouse brain

65% of wounds showed complete healing at 6 months vs. 26% placebo; first gene therapy for a skin disease

Demonstrated gene therapy was approvable but commercially unviable at that time; authorization expired 2017

Phase 3 GlucoGene trial positive; PDUFA March 28, 2026

Rolling BLA submission to FDA; accelerated approval sought

95% reduction in HAE attack rate; some patients completely attack-free for 16+ months after single dose

First patient achieved insulin independence at day 270; subsequent patients showed C-peptide production and reduced insulin requirements

Modest reductions in glycosaminoglycans in some patients; lower than expected editing efficiency; demonstrated in vivo ZFN feasibility

Fatal systemic inflammatory response; led to FDA shutdown of multiple gene therapy trials; established stricter informed consent and conflict-of-interest rules; set back the field by a decade

20-70% enzyme restoration; potentially treats 8,000+ CF, 252,000 Stargardt, 43,500 DMD patients with nonsense mutations

Preclinical development for urea cycle disorder

58% correction in airway cells; pulmonary LNP delivery optimization ongoing

Failed to demonstrate efficacy in post-marketing study; approval withdrawn — cautionary tale for conditional approvals

Phase 1 data showed improved cardiac function markers alongside TTR reduction; Phase 3 ongoing

First patient treated; iPSC-derived heart cells integrated and showed contractile function

Significant TG reduction within 3 days of single low-dose administration

Preclinical data showed >90% reduction in ANGPTL3 and >60% reduction in triglycerides in NHPs

Early clinical data reported dose-dependent reductions in APOC3 and triglycerides

Dose-dependent reductions in PCSK9 protein (up to 84%) and LDL-C (up to 55%); one patient death (pre-existing cardiovascular disease) prompted protocol revision

IND/CTA filing planned H1 2026

Phase 2 enrollment completing mid-2025; readout early 2026

Phase 1 showed 87% overall response rate with no serious adverse events

FDA Fast Track Designation; dual IND approval (US + China)

First patient dosed May 2025

SLE patient in drug-free DORIS remission at Month 6; 4 autoimmune patients treated

7/8 complete remission, durable responses, no serious adverse events

12/13 patients achieved LLDAS; all autoantibodies eliminated; no relapses off medications

Phase 2 BEHOLD study showed visual acuity improvements at 24 weeks but failed to demonstrate durability advantage over anti-VEGF; program discontinued in 2024

FDA IND cleared January 28, 2026. Preclinical: restored vision in aged mice and non-human primate NAION model. First patient enrollment Q1 2026.

FDA IND cleared January 28, 2026. Preclinical: improved visual function and neuronal health in non-human primate NAION injury model.

Published preclinical data showing epigenetic age reversal in mouse tissues; developing multiple delivery platforms for clinical translation

Identified novel reprogramming factor combinations that rejuvenate aged human T cells

Developed mRNA-based reprogramming protocols showing age reversal in human cell models

Published data showing rejuvenation of aged human skin cells and chondrocytes; reduced epigenetic age by up to 25 years in vitro

Preclinical data showed >60% correction of PiZ mutation in non-human primates

IND cleared by FDA in 2024; preclinical data showed sustained glucose homeostasis in GSD1a mice

IND/CTA filing planned H1 2026; initial clinical data expected 2027

IND filing planned mid-2026; initial clinical data expected 2027

100% survival rate over median 7-year follow-up; all patients maintained immune reconstitution

BLA resubmission accepted by FDA

100% survival; >95% of patients off enzyme replacement therapy; no leukemic events with SIN lentiviral vector

All patients showed improved platelet counts and immune function; no leukemic events; most achieved transfusion independence

18 of 20 patients achieved immune reconstitution; 5 developed T-cell leukemia from insertional activation of LMO2 oncogene; 4 of 5 leukemia cases were successfully treated; led to development of safer SIN lentiviral vectors

Partial immune reconstitution; patients still required enzyme replacement therapy but showed improved T cell counts; proved gene therapy concept was viable

Preclinical data showed >70% correction efficiency in patient CD34+ cells; IND-enabling studies underway

FDA approved 2025

First patient dosed in 2022; safety data encouraging; efficacy evaluation ongoing with analytical treatment interruption planned

CCR5-modified T cells persisted for years; one patient had undetectable HIV for 12 weeks off antiretrovirals; proved concept of gene editing for HIV

First patient enrolled in ELIMINATE-B trial 2025; preclinical data showed >95% reduction in HBsAg

First epigenetic editing therapy to enter clinical trials

Patients gained the ability to navigate obstacle courses in low light; improvements sustained at 4+ years

Phase 2b positive; sustained vision gains; BLA filing early 2026

Bilateral visual improvement observed even when only one eye was injected (contralateral effect); EMA MAA filed

Some patients showed clinically meaningful improvements in light sensitivity; 3 of 14 met primary endpoint; program deprioritized in 2023

Preclinical proof-of-concept demonstrated in NHP retinal models

Expands gene therapy access to older SMA patients via intrathecal delivery

IND approved by NMPA; first patient dosed in M.U.S.C.L.E. trial late 2024

First patient completed 6-month follow-up; FDA IND cleared March 2025 for global study

Pig kidney functioned in living human recipient for 2+ months; FDA authorized expanded access

171-day patient survival; 38 days with functional pig liver; published in Nature

Introduced mosaic and off-target edits; CCR5 was not fully knocked out in all cells; sparked global moratorium debate on germline editing; He Jiankui sentenced to 3 years in prison

Claimed telomere lengthening of ~20 years based on leukocyte telomere measurements; results not peer-reviewed; widely criticized by scientific community

VX-880: multiple patients achieved insulin independence; VX-264 eliminates need for immunosuppression