Business
AVROBIO Reports Fourth Quarter and Fiscal Year 2021 Financial Results and Provides Business Update
Provided interim data at WORLDSymposium™ 2022 that showed sustained engraftment across first three patients 1+ year post-gene therapy in Phase 1/2 clinical
About this update from Tectonic Therapeutic, Inc.
[{"type":"text","content":"\nProvided interim data at WORLDSymposium™ 2022 that showed sustained engraftment across first three patients 1+ year post-gene therapy in Phase 1/2 clinical triali for cystinosis; all remain off oral cysteamine to date\n\nInterim clinical data update of AVR-RD-02 in Gaucher disease type 1 planned for 2022; third patient dosed in Phase 1/2 clinical trial\n\nRegulatory interactions planned in 2022 to inform clinical development and registration strategies for programs in Gaucher disease, cystinosis, Hunter syndrome and Pompe disease\n\nStrong balance sheet with cash runway into Q1 2024\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAVROBIO, 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.\n\n“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.”\n\nProgram Updates\n\nPresented interim data from Phase 1/2 clinical trial of AVR-RD-04 in cystinosis at the 18th Annual WORLDSymposium™ 2022:\n\n\nFirst 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.\n\n\nReduction in number of cystine crystals as measured in skin and intestinal mucosa biopsies observed across these three patients.\n\n\nA fourth patient was infus...