CAMBRIDGE, MA.- March 17, 2022– AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today reported financial results for the fourth quarter and year ended Dec. 31, 2021, and provided a business update.
“After a busy 2021 with new patients dosed across three clinical trials, we acted in early January to strategically position AVROBIO for long-term success by reprioritizing our leading lysosomal disorder pipeline. Four weeks after, we reinforced that position by releasing strong interim data from the Phase 1/2 collaborator-sponsored clinical trial for cystinosis at the WORLDSymposiumTM 2022,” said Geoff MacKay, president and CEO of AVROBIO. “We look forward to sharing new data on the three patients dosed to date in our Gaucher disease type 1 program in 2022 and providing updates for our other pipeline programs following meetings with regulatory agencies planned for later this year. We potentially have many exciting catalysts ahead of us and believe that our strong balance sheet will support continued progress through anticipated milestones and into the first quarter of 2024.”
Presented interim data from Phase 1/2 clinical trial of AVR-RD-04 in cystinosis at the 18th Annual WORLDSymposium™ 2022:
- First three patients dosed to date remain off oral cysteamine with follow up durations ranging between 12- and 26-months post-gene therapy infusion. Sustained engraftment has been observed in each of these patients, as evidenced by stable vector copy number (VCN) levels.
- Reduction in number of cystine crystals as measured in skin and intestinal mucosa biopsies observed across these three patients.
- A fourth patient was infused in November 2021.
- No adverse events (AEs) related to the drug product have been reported in the four patients infused to date. All AEs observed have been attributed to myeloablative conditioning, stem cell mobilization, underlying disease or pre-existing conditions.
- Clinical proof-of-concept in adult patients lays the groundwork for potential AVROBIO-sponsored trial planned to begin in 2023.
- The company hosted a conference call providing a full data update.