Skysona

WARNING: HEMATOLOGIC MALIGNANCY Hematologic malignancies, including life-threatening cases of myelodysplastic syndrome and acute myeloid leukemia, have occurred in patients treated with SKYSONA. Patients have been diagnosed between 14 months and 10 years after SKYSONA administration, and the cancers appear to be related to treatment with SKYSONA. Monitor patients closely for evidence of malignancy through complete blood counts at least every 3 months. Monitor patients through assessments for evidence for clonal expansion or predominance at least twice in the first year and annually thereafter; consider bone marrow evaluations as clinically indicated [see Warnings and Precautions (5.1) ] . WARNING: HEMATOLOGIC MALIGNANCY See full prescribing information for complete boxed warning. Hematologic malignancies, including life-threatening cases of myelodysplastic syndrome and acute myeloid leukemia, have occurred in patients treated with SKYSONA. The cancers appear to be the result of treatment with SKYSONA. Monitor patients closely for evidence of malignancy through complete blood counts at least every 3 months and through assessments for evidence for clonal expansion or predominance at least twice in the first year and annually thereafter; consider bone marrow evaluations as clinically indicated. ( 5.1 )

5 WARNINGS AND PRECAUTIONS Serious Infections: Life-threatening bacterial and viral infections may occur. Monitor patients for signs and symptoms of infection. ( 5.2 ) Prolonged Cytopenias: Patients may exhibit cytopenias >1 year after treatment. Monitor patients for bleeding and infection. ( 5.3 ) Delayed Platelet Engraftment: Monitor patients for thrombocytopenia and bleeding until platelet engraftment and count recovery. ( 5.4 ) Risk of Neutrophil Engraftment Failure: Monitor absolute neutrophil counts and if neutrophil engraftment does not occur, give rescue cells. ( 5.5 ) 5.1 Hematologic Malignancy Hematologic malignancies, including myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML), have developed in patients treated with SKYSONA in clinical studies between 14 months and 10 years after SKYSONA administration [see Adverse Reactions (6.1) ] . Malignancies are life-threatening. Death related to treatment for malignancy and relapse of malignancy have occurred . SKYSONA Lenti-D lentiviral vector genomic integration, including into the proto-oncogene MECOM , appears to have mediated the cases of hematologic malignancy. All patients treated with SKYSONA in clinical studies have integrations into MECOM ; however, it is unknown which integrations into MECOM or other genes are likely to lead to malignancy. Consider consultation with hematology experts prior to SKYSONA treatment to inform benefit-risk treatment decision and to ensure adequate monitoring for hematologic malignancy. Consider performing the following baseline hematologic assessments: complete blood count with differential, hematopathology review of peripheral blood smear, and bone marrow biopsy (core and aspirate) with flow cytometry, conventional karyotyping, and next generation sequencing (NGS) with a molecular panel appropriate for age and including coverage for gene mutations expected in myeloid and lymphoid malignancies; and testing for germline mutations that are associated with hematologic malignancy. Early diagnosis of hematologic malignancy can be critically important, therefore, monitor patients treated with SKYSONA lifelong for hematologic malignancy. For at least the first fifteen years after treatment with SKYSONA, monitor via complete blood count (with differential) at least every 3 months and via integration site analysis or other testing for evidence of clonal expansion and predominance at least twice in the first year and then annually. Consider appropriate expert consultation and additional testing such as more frequent complete blood count (with differential) and integration site analysis, bone marrow studies, and gene expression studies in the following settings after treatment with SKYSONA: Delayed or failed engraftment of platelets or other cell lines (while all patients are at risk for hematologic malignancy, patients who do not achieve unsupported platelet counts of ≥ 20 × 10 9 /L on or after Day 60 appear to be at higher risk); or New or prolonged cytopenias; or, Presence of clonal expansion or predominance (e.g., increasing relative frequency of an integration site, especially if ≥ 10% and present in MECOM or another proto-oncogene known to be involved in hematologic malignancy). If hematologic malignancy is detected in a patient who received SKYSONA, contact Genetix Biotherapeutics at 1-833-999-6378 for reporting and to obtain instructions on collection of samples for further testing. Post-Marketing Long Term Follow-Up Study Patients who intend to receive treatment with SKYSONA are encouraged to enroll in the study, as available, to assess the long-term safety of SKYSONA and the risk of malignancies occurring after treatment with SKYSONA by calling Genetix Biotherapeutics, Inc. at 1-833-999-6378. The study includes monitoring (at pre-specified intervals) for clonal expansion. 5.2 Serious Infections Severe infections, including life-threatening and fatal infections, have occurred in patients after SKYSONA infusion. Febrile neutropenia was commonly observed in clinical studies and may be a sign of a serious infection. In the event of febrile neutropenia, evaluate for infection and manage with broad-spectrum antibiotics, fluids, and other supportive care as medically indicated. Monitor patients for signs and symptoms of infection before and after SKYSONA administration and treat appropriately. Administer prophylactic antimicrobials according to best clinical practices and clinical guidelines. Avoid administration of SKYSONA in patients with active infections. 5.3 Prolonged Cytopenias Patients may exhibit cytopenias, including pancytopenia, for > 1 year following conditioning and SKYSONA infusion [see Adverse Reactions (6.1) ] . Monitor blood counts until normalization and assess patients for signs and symptoms of bleeding and/or infection prior to and after SKYSONA administration. 5.4 Delayed Platelet Engraftment Delayed platelet engraftment (platelet count ≤ 50 × 10 9 /L beyond 60 days after treatment with SKYSONA) has been observed [see Adverse Reactions (6.1) ] . Bleeding risk is increased prior to platelet engraftment and may continue after engraftment in patients with prolonged thrombocytopenia. Patients should be made aware of the risk of bleeding until platelet recovery has been achieved. Monitor patients for thrombocytopenia and bleeding according to standard guidelines. Conduct frequent platelet counts until platelet engraftment and platelet recovery are achieved. Perform blood cell count determination and other appropriate testing whenever clinical symptoms suggestive of bleeding arise. 5.5 Risk of Neutrophil Engraftment Failure There is a potential risk of neutrophil engraftment failure after treatment with SKYSONA. Neutrophil engraftment failure was defined as failure to achieve 3 consecutive absolute neutrophil counts (ANC) ≥ 0.5 × 10 9 cells/L obtained on different days by Day 43 after infusion of SKYSONA. Monitor neutrophil counts until engraftment has been achieved. If neutrophil engraftment failure occurs in a patient treated with SKYSONA, provide rescue treatment with the back-up collection of CD34+ cells [see Adverse Reactions (6.1) ] . 5.6 Hypersensitivity Reactions Allergic reactions may occur with the infusion of SKYSONA. The dimethyl sulfoxide (DMSO) in SKYSONA may cause hypersensitivity reactions, including anaphylaxis which is potentially life-threatening and requires immediate intervention. 5.7 Anti-retroviral Use Patients should not take anti-retroviral medications for at least one month prior to mobilization or the expected duration for elimination of the medications, and until all cycles of apheresis are completed. Anti-retroviral medications may interfere with manufacturing of the apheresed cells [see Drug Interactions (7.2) ] . If a patient requires anti-retrovirals for HIV prophylaxis, mobilization and apheresis of CD34+ cells should be delayed until HIV infection is adequately ruled out. 5.8 Laboratory Test Interference SKYSONA affects polymerase chain reaction (PCR) assays for HIV due to LVV provirus insertion. A PCR-based assay should not be used to screen for HIV infection in patients treated with SKYSONA as a false-positive test result is likely.

6 ADVERSE REACTIONS Most common non-laboratory adverse reactions (≥ 20%): mucositis, nausea, vomiting, febrile neutropenia, alopecia, decreased appetite, abdominal pain, constipation, pyrexia, diarrhea, headache, rash. ( 6.1 ) Most common Grade 3 or 4 laboratory abnormalities (≥ 40%): leukopenia, lymphopenia, thrombocytopenia, neutropenia, anemia, hypokalemia. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Genetix Biotherapeutics at 1-833-999-6378 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The safety data described in this section reflect exposure to a single dose of SKYSONA in 67 patients with CALD. Data were obtained from two single-arm trials and, for 36 patients, from a long-term follow-up study [see Clinical Studies (14) ] . The median (min, max) age across studies was 6 (4, 14) years; 100% were male; 54% were White/Caucasian, 4% were Black or African American, 1% were Asian, 10% were of other races including mixed race, and 30% did not report race; 25% were of Hispanic ethnicity. The median (min, max) duration of follow-up at time of initial approval was 24 (1, 88) months. In the two trials, serious adverse reactions from Day 1 (SKYSONA infusion) to last follow-up occurred in 54% of patients. The most common non-laboratory, serious adverse reactions (≥ 3% incidence) that occurred after treatment with SKYSONA were febrile neutropenia (18%), pyrexia (fever) (18%), seizure (7%), myelodysplastic syndrome (4%), pseudomonal bacteremia (3%), pancytopenia (3%), vascular device infection (3%), mucositis (3%), and vomiting (3%). Most common non-laboratory adverse reactions by time of onset follow: During mobilization and conditioning and occurring in ≥ 20% of patients: nausea (79%), vomiting (72%), decreased appetite (42%), catheter site pain (39%), constipation (30%), headache (24%), abdominal pain (21%), rash (13%) In the first 60 days after treatment with SKYSONA in ≥ 15% of patients: mucositis (88%), febrile neutropenia (73%), alopecia (72%), abdominal pain (33%), vomiting (31%), decreased appetite (31%), pyrexia (27%), nausea (27%), constipation (21%), diarrhea (21%), epistaxis (19%), pruritus (18%), headache (16%), oropharyngeal pain (16%), skin hyperpigmentation (16%), anxiety (15%) Between 60 days and 1 year after treatment with SKYSONA in ≥ 5% of patients: pyrexia (fever) (9%) and vomiting (6%) Table 1 presents non-laboratory adverse reactions reported in at least 10% of patients between the start of conditioning and 24 months after SKYSONA administration. Table 2 presents Grade 3 or 4 laboratory abnormalities that occurred in at least 40% of patients between the start of conditioning and 24 months after SKYSONA administration. Table 1: Non-Laboratory Adverse Reactions Reported in ≥ 10% of Patients Between the Start of Conditioning and 24 Months Following Treatment with SKYSONA Includes adverse events associated with conditioning. Adverse Reaction Any Grade n (%) Grade 3 or Higher n (%) Blood and lymphatic system disorders -- -- Febrile neutropenia Febrile neutropenia includes febrile bone marrow aplasia and febrile neutropenia. 49 (73%) 49 (73%) Cardiac disorders -- -- Tachycardia Tachycardia includes sinus tachycardia and tachycardia. 10 (15%) 0 Eye disorders -- -- Vision blurred 7 (10%) 0 Gastrointestinal disorders -- -- Mucositis Mucositis includes anal inflammation, colitis, gastrointestinal inflammation, mucosal inflammation, proctitis, and stomatitis. Encompasses more than one system organ class. 62 (92%) 34 (51%) Nausea 56 (84%) 17 (25%) Vomiting 51 (76%) 12 (18%) Abdominal pain Abdominal pain includes abdominal discomfort, abdominal pain, and abdominal pain upper. 30 (45%) 2 (3%) Constipation 28 (42%) 0 Diarrhea 19 (28%) 1 (1%) General disorders and administration site conditions -- -- Pyrexia 24 (36%) 3 (4%) Injury, poisoning and procedural complications -- -- Transfusion reaction Transfusion reaction includes allergic transfusion reaction and anaphylactic transfusion reaction. 8 (12%) 2 (3%) Metabolism and nutrition disorders -- -- Decreased appetite 43 (64%) 27 (40%) Nervous system disorders -- -- Headache 19 (28%) 0 Anxiety Anxiety includes akathisia, agitation, anxiety, and irritability. 10 (15%) 0 Respiratory, thoracic, and mediastinal disorders -- -- Epistaxis 13 (19%) 5 (7%) Oropharyngeal pain Oropharyngeal pain includes mouth ulceration, oral pain, and oropharyngeal pain. 12 (18%) 3 (4%) Cough 7 (10%) 0 Skin and subcutaneous tissue disorders -- -- Alopecia 48 (72%) 1 (1%) Rash Rash includes rash, rash erythematous, rash maculo-papular, and urticaria. 14 (21%) 0 Pruritus Pruritus includes anal pruritus, pruritus, and pruritus allergic. 13 (19%) 0 Skin hyperpigmentation 12 (18%) 0 Vascular disorders -- -- Hypertension 8 (12%) 1 (1%) Hematologic Malignancy As of July 2025, hematologic malignancies have been diagnosed in 10/67 (15%) clinical study patients [see Warnings and Precautions (5.1) ] . At diagnosis of hematologic malignancy, all 10 patients had predominant integrations; 7 in proto-oncogenes, including 6 in MECOM . Pathological diagnoses ranged between myelodysplastic syndrome (MDS)-unilineage dysplasia to acute myeloid leukemia. As of July 2025, 9 of the 10 patients had received allogeneic hematopoietic stem cell transplant. One of the patients with MDS relapsed 6 months after an allogeneic transplant and required re-treatment of MDS. Serious Infections Important opportunistic infections that have been diagnosed within the first 3 months after treatment with SKYSONA include BK cystitis, cytomegalovirus reactivation, human herpesvirus-6 viremia, candidiasis, and bacteremias. Opportunistic infections after the first 3 months include an atypical mycobacterium vascular device infection, pseudomonas bacteremia, and Epstein-Barr virus reactivations diagnosed as late as 18 months after treatment with SKYSONA. Serious infections involving adenovirus include a case of transverse myelitis at 6 months that was attributed to adenovirus and entero/rhinovirus infection, and a fatal adenovirus infection at 21 months in a patient with CALD progression who developed multisystem organ failure. Grade 3 or higher infections occurred in 21% of all patients (12% bacterial, 3% viral, and 6% unspecified). The most common Grade 3 or higher infections were vascular device infections (7% of patients) diagnosed as late as 6 months after treatment with SKYSONA, and bacteremias (6% of patients) diagnosed as late as 8 months after treatment with SKYSONA. Table 2: Grade 3 or 4 Laboratory Abnormalities Occurring in ≥ 40% of Patients Between the Start of Conditioning and 24 Months Following Treatment with SKYSONA Includes laboratory abnormalities associated with conditioning. Laboratory Abnormality Grade 3 or 4 n (%) Leukopenia 67 (100%) Lymphopenia 67 (100%) Thrombocytopenia 67 (100%) Neutropenia 64 (96%) Anemia 56 (84%) Hypokalemia 28 (42%) Cytopenias Grade 3 or higher cytopenias on or after Day 60 following SKYSONA infusion occurred in 47% of patients and included low platelet count (14%), low neutrophil count (22%), low lymphocyte count (27%), and low hemoglobin (2%). Grade 3 cytopenias persisted beyond Day 100 in 15% of patients and included low platelet count (7%), low neutrophil count (9%), and low lymphocyte count (6%). Serious adverse reactions of pancytopenia occurred in two patients who required support with blood and platelet transfusions as well as growth factors (G-CSF for up to 6 months and eltrombopag for up to 14 months) after SKYSONA administration. One patient had intercurrent parvovirus infection and his pancytopenia was ongoing at least two years after SKYSONA administration. Pancytopenia in the other patient was ongoing until he was diagnosed with myelodysplastic syndrome approximately two years after SKYSONA administration. Platelet Engraftment Delay Platelet engraftment was defined as 3 consecutive platelet values ≥ 20 × 10 9 /L on different days and no platelet transfusions administered for 7 days immediately preceding and during the evaluation period. Platelet engraftment was not achieved by Day 43 after SKYSONA administration in 13 of 63 patients (21%). Patients treated with SKYSONA achieved platelet engraftment at median (min, max) Day 29 (14, 108) in clinical studies, including two patients treated with a thrombopoietin receptor agonist at the time engraftment criteria were met until 10 or 14 months after treatment with SKYSONA. One of the two had persistence of mild thrombocytopenia after discontinuation of the eltrombopag, and the other remained severely thrombocytopenic (platelet count < 50 × 10 9 /L) until he was diagnosed with myelodysplastic syndrome approximately 2 years after SKYSONA administration [see Warnings and Precautions (5.4) ] . Neutrophil Engraftment Neutrophil engraftment was defined as achieving 3 consecutive absolute neutrophil counts (ANC) ≥ 0.5 × 10 9 cells/L (after initial post-infusion nadir) obtained on different days by Day 43 after SKYSONA infusion. While all patients met criteria for neutrophil engraftment following treatment with SKYSONA in clinical trials, 7 of 67 patients (10%) required G-CSF beyond Day 43, including 3 patients who required G-CSF more than 3 months after treatment with SKYSONA. In three other patients, G-CSF discontinuation was followed by a decrease in neutrophil count to < 0.5 × 10 9 cells/L occurring within 3 days and lasting for two to five weeks [see Warnings and Precautions (5.5) ] . Infusion-Related Reactions Nausea and vomiting have occurred on the day of infusion. Premedication with anti-emetic, anti-pyretics and/or antihistamines may be considered.

<table width="80%"><caption>Table 1: Non-Laboratory Adverse Reactions Reported in &#x2265; 10% of Patients Between the Start of Conditioning and 24 Months Following Treatment with SKYSONA<footnote>Includes adverse events associated with conditioning.</footnote></caption><col width="55%" align="left" valign="top"/><col width="20%" align="center" valign="top"/><col width="25%" align="center" valign="top"/><thead><tr><th styleCode="Lrule Rrule">Adverse Reaction</th><th styleCode="Rrule">Any Grade n (%)</th><th styleCode="Rrule">Grade 3 or Higher n (%)</th></tr></thead><tbody><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Blood and lymphatic system disorders</content></td><td styleCode="Rrule">--</td><td styleCode="Rrule">--</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Febrile neutropenia<footnote>Febrile neutropenia includes febrile bone marrow aplasia and febrile neutropenia.</footnote></td><td styleCode="Rrule">49 (73%)</td><td styleCode="Rrule">49 (73%)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Cardiac disorders</content></td><td styleCode="Rrule">--</td><td styleCode="Rrule">--</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Tachycardia<footnote>Tachycardia includes sinus tachycardia and tachycardia.</footnote></td><td styleCode="Rrule">10 (15%)</td><td styleCode="Rrule">0</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Eye disorders</content></td><td styleCode="Rrule">--</td><td styleCode="Rrule">--</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Vision blurred</td><td styleCode="Rrule">7 (10%)</td><td styleCode="Rrule">0</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Gastrointestinal disorders</content></td><td styleCode="Rrule">--</td><td styleCode="Rrule">--</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Mucositis<footnote>Mucositis includes anal inflammation, colitis, gastrointestinal inflammation, mucosal inflammation, proctitis, and stomatitis.</footnote><footnote ID="ft1">Encompasses more than one system organ class.</footnote></td><td styleCode="Rrule">62 (92%)</td><td styleCode="Rrule">34 (51%)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Nausea</td><td styleCode="Rrule">56 (84%)</td><td styleCode="Rrule">17 (25%)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Vomiting</td><td styleCode="Rrule">51 (76%)</td><td styleCode="Rrule">12 (18%)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Abdominal pain<footnote>Abdominal pain includes abdominal discomfort, abdominal pain, and abdominal pain upper.</footnote></td><td styleCode="Rrule">30 (45%)</td><td styleCode="Rrule">2 (3%)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Constipation</td><td styleCode="Rrule">28 (42%)</td><td styleCode="Rrule">0</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Diarrhea</td><td styleCode="Rrule">19 (28%)</td><td styleCode="Rrule">1 (1%)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">General disorders and administration site conditions</content></td><td styleCode="Rrule">--</td><td styleCode="Rrule">--</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Pyrexia</td><td styleCode="Rrule">24 (36%)</td><td styleCode="Rrule">3 (4%)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Injury, poisoning and procedural complications</content></td><td styleCode="Rrule">--</td><td styleCode="Rrule">--</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Transfusion reaction<footnote>Transfusion reaction includes allergic transfusion reaction and anaphylactic transfusion reaction.</footnote></td><td styleCode="Rrule">8 (12%)</td><td styleCode="Rrule">2 (3%)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Metabolism and nutrition disorders</content></td><td styleCode="Rrule">--</td><td styleCode="Rrule">--</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Decreased appetite</td><td styleCode="Rrule">43 (64%)</td><td styleCode="Rrule">27 (40%)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Nervous system disorders</content></td><td styleCode="Rrule">--</td><td styleCode="Rrule">--</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Headache</td><td styleCode="Rrule">19 (28%)</td><td styleCode="Rrule">0</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Anxiety<footnote>Anxiety includes akathisia, agitation, anxiety, and irritability.</footnote><footnoteRef IDREF="ft1"/></td><td styleCode="Rrule">10 (15%)</td><td styleCode="Rrule">0</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Respiratory, thoracic, and mediastinal disorders</content></td><td styleCode="Rrule">--</td><td styleCode="Rrule">--</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Epistaxis</td><td styleCode="Rrule">13 (19%)</td><td styleCode="Rrule">5 (7%)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Oropharyngeal pain<footnote>Oropharyngeal pain includes mouth ulceration, oral pain, and oropharyngeal pain.</footnote><footnoteRef IDREF="ft1"/></td><td styleCode="Rrule">12 (18%)</td><td styleCode="Rrule">3 (4%)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Cough</td><td styleCode="Rrule">7 (10%)</td><td styleCode="Rrule">0</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Skin and subcutaneous tissue disorders</content></td><td styleCode="Rrule">--</td><td styleCode="Rrule">--</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Alopecia</td><td styleCode="Rrule">48 (72%)</td><td styleCode="Rrule">1 (1%)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Rash<footnote>Rash includes rash, rash erythematous, rash maculo-papular, and urticaria.</footnote></td><td styleCode="Rrule">14 (21%)</td><td styleCode="Rrule">0</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Pruritus<footnote>Pruritus includes anal pruritus, pruritus, and pruritus allergic.</footnote><footnoteRef IDREF="ft1"/></td><td styleCode="Rrule">13 (19%)</td><td styleCode="Rrule">0</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Skin hyperpigmentation</td><td styleCode="Rrule">12 (18%)</td><td styleCode="Rrule">0</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Vascular disorders</content></td><td styleCode="Rrule">--</td><td styleCode="Rrule">--</td></tr><tr><td styleCode="Lrule Rrule">Hypertension</td><td styleCode="Rrule">8 (12%)</td><td styleCode="Rrule">1 (1%)</td></tr></tbody></table>

<table width="90%"><caption>Table 2: Grade 3 or 4 Laboratory Abnormalities Occurring in &#x2265; 40% of Patients Between the Start of Conditioning and 24 Months Following Treatment with SKYSONA<footnote>Includes laboratory abnormalities associated with conditioning.</footnote></caption><col width="50%" align="left" valign="middle"/><col width="50%" align="center" valign="middle"/><thead><tr><th styleCode="Lrule Rrule" valign="top">Laboratory Abnormality</th><th styleCode="Rrule">Grade 3 or 4 n (%)</th></tr></thead><tbody><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Leukopenia</td><td styleCode="Rrule">67 (100%)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Lymphopenia</td><td styleCode="Rrule">67 (100%)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Thrombocytopenia</td><td styleCode="Rrule">67 (100%)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Neutropenia</td><td styleCode="Rrule">64 (96%)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Anemia</td><td styleCode="Rrule">56 (84%)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Hypokalemia</td><td styleCode="Rrule">28 (42%)</td></tr></tbody></table>