Orchard Therapeutics has begin proof of concept trials for two separate rare diseases for which the current standard of care is quite poor.
The first therapy is called OTL-103, and it is an autologous hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome (WAS). WAS is a life threatening immune disorder and the current standard of care is a bone marrow transplant, which carries a high risk for mortality.
Orchard Therapeutics seeks to enroll 6 patients for an early trial of it’s WAS treatment. At this time, they have only dosed one patient. Prospective patients can find more information at clinicaltrials.gov. More information about OTL-103 can be found in Orchard Therapeutic’s press release.
The second therapy is called OTL-300, and is a treatment aimed at blood transfusion-dependent Beta Thalassemia (BA) patients. Like OTL-103, it is a stem cell based gene therapy that seeks to target the underlying disorder.
In an early clinical proof-of-concept trial involving 9 patients, the results were extremely promising:
- All nine patients met the safety endpoint of survival with follow-up ranging from 16 to 43 months (3.6 years). No adverse events related to the product were reported.
- Vector integration profiles had the expected polyclonal genomic distribution seen with other lentiviral vector mediated HSC gene therapy studies, and no evidence of abnormal proliferation or clonal dominance was observed.
- The primary efficacy endpoint of transfusion
reduction at 12 months following treatment was achieved in eight of nine
TDT patients treated with OTL-300.
– Of the six pediatric patients treated, four achieved transfusion independence and one showed a reduction in transfusion requirement.
– All three adult patients had a reduction in their transfusion requirements.
– As published previously in Nature Medicine, one pediatric patient did not have a reduced transfusion requirement compared to pre-treatment levels at 12 months, which was attributed to poor engraftment of the gene-modified cells.
For more information about OTL-300, please see Orchard Therapeutics’ press release.