AVROBIO, Inc. - AVRO STOCK NEWS

AVROBIO, Inc (Nasdaq: AVRO) announced that the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) granted an Innovation Passport to its investigational gene therapy, AVR-RD-02, for treating Gaucher disease. This status is part of the Innovative Licensing and Access Pathway (ILAP), aimed at expediting regulatory review and patient access. The company plans to provide interim clinical data for its Phase 1/2 trial by Q4 2022, with an estimated 30,000 patients affected globally. This is a significant step towards early patient treatment in the U.K.

AVROBIO has reported positive data on the combined use of two advanced safety assays designed to evaluate genotoxicity risk in gene therapy vectors. The assays, developed in collaboration with Professor Axel Schambach, aim to identify safer vectors for hematopoietic stem cell gene therapy before clinical trials. Notably, no cases of insertional oncogenesis have been observed in AVROBIO's clinical programs to date. At the 29th Annual ESGCT Congress, the findings were presented, highlighting the potential for safer gene therapies and the importance of early risk assessment.

AVROBIO, Inc. (NASDAQ: AVRO), a clinical-stage gene therapy company, has granted 28,000 restricted stock units (RSUs) to two new employees as inducement awards under the 2019 Inducement Plan. The RSUs were issued on Oct. 3, 2022, and vest over four years, with 25% vesting on the first anniversary of the new hire dates. This step is part of AVROBIO's strategy to attract top talent to advance its mission of developing gene therapies for genetic diseases such as cystinosis and Gaucher disease.

AVROBIO has received the FDA's rare pediatric disease designation for its investigational gene therapy AVR-RD-04, aimed at treating cystinosis, a severe genetic disorder. This designation is part of the FDA's initiative to expedite drug development for rare diseases. AVR-RD-04 genetically modifies patients’ stem cells to produce cystinosin, a deficient protein. Preliminary Phase 1/2 trial data indicates the therapy is well-tolerated with mild adverse events, potentially benefiting multiple organ systems. Furthermore, the therapy received orphan drug designation from FDA and EMA.

AVROBIO has received approval from the U.K. MHRA for a Phase 1/2 clinical trial of AVR-RD-05, a gene therapy targeting Hunter syndrome in infants aged 3 to 12 months. The trial, conducted in collaboration with the University of Manchester, aims to assess the safety and tolerability of this innovative autologous hematopoietic stem cell therapy. The FDA has previously granted rare pediatric disease and orphan drug designations for AVR-RD-05. The first patient is expected to be dosed in 1H 2023, marking a significant step forward in treating this debilitating condition.

AVROBIO, a clinical-stage gene therapy company, announced the grant of 58,000 restricted stock units (RSUs) to three new employees as inducement awards under its 2019 Inducement Plan. This action, compliant with Nasdaq Listing Rule 5635(c)(4), aims to incentivize new hires. The RSUs are set to vest over four years, with a quarter vesting on the first anniversary and the remainder in equal installments annually. AVROBIO focuses on developing personalized gene therapies targeting genetic diseases.

AVROBIO, a clinical-stage gene therapy company, announced its participation in four investor conferences scheduled between September 6-14, 2022. These include Citi's BioPharma Conference, Wells Fargo Healthcare Conference, H.C. Wainwright Global Investment Conference, and Morgan Stanley's Global Healthcare Conference. AVROBIO aims to revolutionize treatment for genetic diseases through its proprietary plato® gene therapy platform, targeting conditions such as cystinosis and Gaucher disease. Presentations will be available on their website, and archived for 90 days.

AVROBIO, a clinical-stage gene therapy company, presented positive early data from its Phase 1/2 cystinosis trial at the ASGCT Annual Meeting, indicating gene therapy's potential to stabilize or improve symptoms. All five patients treated remain off oral cysteamine post-treatment. AVROBIO reported a net loss of $28.1 million for Q2 2022, a decrease from $31.4 million in Q2 2021, partly due to reduced R&D expenses, with cash reserves of $132.4 million expected to fund operations into Q1 2024. The company plans upcoming regulatory interactions and updates on various therapies.