Exhibit 99.2
ASGCT 2023 cystinosis update
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ASGCT 2023 cystinosis update 3 Summary of key points Continued positive
trends across multiple biomarkers and neurocognitive measures seen in Phase 1/2 collaborator-sponsored trial All patients remain off oral cysteamine, up to 36 months post-gene therapy Safety and tolerability profile remains
strong Positive interactions with U.K. Medicines and Healthcare products Regulatory Agency (MHRA) and U.S. Food and Drug Administration (FDA) in Q1 2023
Phase 1/2 Objectives Patients Safety and tolerability Hypothesis generation
of clinical efficacy endpoints 6 patients Adults and adolescents Cohorts 1-2 >18 years; Cohort 3 >14 years Male and female Oral and ophthalmic cysteamine Cystinosis Phase 1/2 dosing complete 4 Clinical trial funded in part by
grants to UCSD from the California Institute for Regenerative Medicine (CIRM), Cystinosis Research Foundation (CRF) and National Institutes of Health (NIH) Collaborator-sponsored University of California, San Diego
VCN: Vector Copy Number; PBCs: Peripheral Blood Cells; dg: Diploid
Genome *Average of 2 drug products Note: Patient 2 was lost to follow-up after 12 months Drug Product VCN/dg Patient 1 2.1 Patient 2 1.3 Patient 3 1.6 Patient 4 0.6 Patient 5 2.5 Patient 6 2.9* VCN trending as expected,
indicating sustained engraftment CYSTINOSIS PHASE 1/2: PATIENTS 1-6 NEW DATA POINT 5
Therapeutic range is <1.0 Half Cystine (nmol/mg protein). Measure of 1 is
level of healthy heterozygote.; For Patient 1, Leukocyte Cystine Quantification was initiated at approximately week 20 ; *Patient 1: Hemolyzed sample which may potentially lead to lower results; mo=month; Note: Patient 2 was lost to follow-up
after 12 months NEW DATA 6 Sustained leukocyte cystine level reduction CYSTINOSIS PHASE 1/2: PATIENTS 1-6 Lift updated data from next slide and put on this slide NEW DATA POINT *
7 Skin and gastrointestinal mucosa cystine crystal reduction CYSTINOSIS PHASE
1/2: PATIENTS 1-5 Rectal Biopsy Average intracytoplasmic crystals per cell Skin Biopsy 8.4 Put on AVRO slide in AVRO format Note: Patient 2 was lost to follow-up after 12 months NEW DATA POINT
Improvement or stabilization in motor coordination and visual perception 8 The
Beery – Buktenica Developmental Test of Visual Motor Integration (Beery VMI) [6th edition] is a standardized test evaluating the ability of the brain to interpret and translate visual information into an exact motor response; Patient 2 did
not complete the examination CYSTINOSIS PHASE 1/2: PATIENTS 1-5 Patient 1 Patient 3 Patient 4 Patient 5 NEW DATA POINT
All patients continue to be oral cysteamine-independent 9 Patient #1 out 3
years CYSTINOSIS PHASE 1/2: PATIENTS 1-6 Patient Months off cysteamine pills and eye drops post Ctns-rd-04 infusion Current status Cysteamine Pills Patient 1 OFF Patient 2 Lost to follow-up Patient 3 OFF Patient
4 OFF Patient 5 OFF Patient 6 OFF Cysteamine Eye Drops Patient 1 OFF Patient 2 Lost to follow-up Patient 3 OFF Patient 4 Was not on cysteamine eye drops prior to infusion OFF Patient 5 OFF Patient 6 OFF Note: Patients 2, 3
and 5 stopped cysteamine eye drops 1-month post-transplant (per protocol); Patient 1 stopped cysteamine eye drops prior to baseline; Data as of May 8, 2023. Patient 2 has elected not to return since the 12-month follow-up visit. NEW DATA
POINT
eGFR: Estimated Glomerular Filtration Rate; eGFR calculated using CKD-EPI
formula Note: Patient 2 was lost to follow-up after 12 months 10 eGFR data reinforce need for early intervention CYSTINOSIS PHASE 1/2: PATIENT 1-6 Lift updated data from next slide and put on this slide CYSTINOSIS PHASE 1/2: PATIENTS
1-5
No adverse events related to drug product 11 No SAEs or AEs related to drug
product AE: Adverse Event; SAE: Serious Adverse Event No SAEs reported Preliminary AEs reported (as of May 8, 2023) N=46 for patient 1; N=22 for patient 2; N=8 for patient 3; N=29 for patient 4; N=37 for patient 5; N=41 for patient
6 Majority of AEs are mild or moderate 1 severe AE for subject 1 Appendicitis (resolved) – unrelated to study treatment or procedures AEs are generally consistent with myeloablative conditioning, study procedures, underlying disease or
co-morbid or pre-existing conditions: Pre-gene therapy treatment and prior to conditioning (not all events listed) Diarrhea, hypokalemia, hypomagnesemia, thrombocytopenia, dizziness, dehydration, vomiting, bone pain,
headache Post-treatment (not all events listed) Pancytopenia, deep vein thrombosis, Staphylococcus sepsis, Coronavirus infection, alopecia, rash, mucositis Intermittent: diarrhea, vomiting, loss of appetite, epistaxis, blurry vision,
febrile neutropenia, hypomagnesemia, hypokalemia No adverse events related to drug product
ASGCT 2023 cystinosis update