Sarepta publishes positive data for gene therapy SRP-9001 and SRP-9003 with project reports.

Sarepta Therapeutics is a leader in precision genetic medicine for rare diseases.
, the company recently announced positive clinical results for two gene therapies, SRP-9001 and SRP-9003.
SRP-9001 SRP-9001 is a gene therapy for Duchy muscular dystrophy (DMD) designed to transfer genes that encode micro-dystrophin to muscle tissue, allowing muscle cells to express micronourished proteins.
the results included two-year follow-up data for four non-bedridden patients (4-7 years old).
results show that polar mobility (NSAA) assessments continued to improve over baseline after two years of one-time injections of SRP-9001.
90th day, vector transductors were confirmed in all patients, and NSAA scores showed improved functionality and lower crea acid kinase (CK) levels.
patients were treated by an average of 5.5 points more than the baseline and 7.0 points more than the baseline two years after treatment.
2 years, all patients had good tolerance to SRP-9001.
all adverse events are mild or moderate and occur within 90 days of treatment.
no serious adverse events or signs of complement activation.
, these patients are now 6-8 years old.
analysis of DMD historical data published last year in the journal PLOS One found that, on average, DMD scores for 6-8 year olds peak at 6.3 years of age and then begin to decline.
two-year results released this week show that SRP-9001 single injection therapy has changed the course of the disease.
SRP-9003 SRP-9003 is a gene therapy for leg-band muscular dystrophy type 2E (LGMD2E) designed to transfer genes that encode full-length β-myoglobulin to muscle tissue, allowing muscle cells to express β-myoglobin.
the results included 18-month functional data for 3 patients in a low-dose queue in clinical trials and 6 months function data for 3 patients in a high-dose queue.
results showed that after infusion of SRP-9003 in two dose groups, effective transductivity of skeletal muscle and expression of β-myosin were observed, and creatinase (CK) was significantly reduced during 90 days of treatment.
queue specificity results were as follows: - Queue 1 (low dose), 18 months: All 3 patients continued to show improvements in baseline levels in all functional measurements, including muscular dystrophy assessment (NSAD), start time, fourth-order climb, 100-meter walking test, 10-meter walking test.
NSAD improved by an average of 3.0 in six months and 5.7 at 18 months compared to the baseline.
since the release of the one-year update in June 2020, no new drug-related safety signals have been observed, nor have there been any signs of plate plateboard counts falling outside the normal range or complement activation.
- Queue 2 (high dose), 6 months: All 3 patients showed improvement in all functional indicators, including NSAD, start time, fourth-order climb, 100m walking test, 10m walking test.
nsaD improved by an average of 3.7 compared to baseline checks.
since the results of protein expression were published in June 2020, no new drug-related safety signals have been observed, and no signs of plate plateboard counts falling outside the normal range or complement activation have been observed.
results confirmed that improvements in functional indicators in 2 groups at 18 and 6 months were significantly different from those predicted in the age-matching natural history group.
the persistence of this functional outcome response strengthens the SRP-9003's entry into the muscles and suggests that it is improving disease-mediated muscle damage.
source: New data bolsters Sarepta gene therapies for two deadly muscle diseases.