| Over a month ago | ||||
BTIG analyst Yun Zhong… BTIG analyst Yun Zhong upgraded Avrobio to Buy from Neutral with a $4 price target. This morning's update, from four Gaucher disease type 1 patients that received AVR-RD-02 treatment in the ongoing Phase 1/2 Guard1 study and one type 3 patient treated on named-patient basis in the UK, support the company's lentiviral gene therapy programs' promising potential in lysosomal storage disorders, according to the analyst. The Gaucher data, although still from a limited number of patients and after modest follow-up periods, "still justify the re-inclusion of the program into valuation" and, hence, the upgrade to Buy. | ||||
Shares of Avrobio are up … Shares of Avrobio are up 6.10% or 5c to 89c per share in pre-market trading. | ||||
AVROBIO announced new… AVROBIO announced new interim pharmacokinetic, pharmacodynamic and clinical efficacy data, showing stabilization or reversal of multiple clinically relevant measures in five patients with Gaucher disease after they received a single dose of AVR-RD-02, an investigational hematopoietic stem cell gene therapy. In addition, following positive discussions with regulators, AVROBIO plans to initiate a global, registrational Phase 2/ 3 clinical trial in Gaucher disease type 3 in the second half of 2023..."We are thrilled to share new, compelling data from patients impacted across the spectrum of Gaucher disease, the most common lysosomal disorder. This includes what we believe to be transformational data from the first pediatric GD3 patient treated with an HSC gene therapy, showing complete biochemical correction, which means both enzyme activity and substrate levels have normalized post gene therapy. This pharmacodynamic efficacy equates with improvements in major refractory elements of disease for this patient, something the child has never experienced on current standard of care," said Essra Ridha, M.D., MRCP, FFPM, chief medical officer at AVROBIO. "Following constructive regulatory conversations, including with FDA, we are now focused on initiating a randomized controlled, Phase 2/3 clinical trial for GD3 next year, the first such trial for a gene therapy, to further evaluate the benefit-risk profile of AVR-RD-02 in a clinical trial setting." AVROBIO believes its plato gene therapy platform is late stage-trial ready, with no major CMC changes anticipated | ||||
Management host a virtual… Management host a virtual Gaucher Disease Program Update for analysts and investors on December 7 at 8 am. Webcast Link | ||||
Management host a virtual… Management host a virtual Gaucher Disease Program Update for analysts and investors on December 7 at 8 am. Webcast Link | ||||
Management host a virtual… Management host a virtual Gaucher Disease Program Update for analysts and investors on December 7 at 8 am. Webcast Link | ||||
As of September 30 ,… As of September 30 , AVROBIO had $116M in cash and cash equivalents, as compared to $189.6M in cash and cash equivalents as of December 31, 2021. Based on AVROBIO's current operating plan, AVROBIO expects its cash and cash equivalents as of September 30 , will enable AVROBIO to fund its operating expenses and capital expenditure requirements into the first quarter of 2024. ."We had a steady cadence of news this quarter highlighting our steadfast efforts to bring our gene therapies to patients," said Geoff MacKay, president and CEO of AVROBIO. "We look forward to providing a comprehensive update next month on our HSC gene therapy targeting Gaucher disease, the most common lysosomal disorder, impacting an estimated 30,000 patients worldwide. This update will contain interim clinical data from our Gaucher disease type 1 Phase 1/2 trial, including data out over two years. Notably, we plan to provide an update on our regulatory interactions for a planned Gaucher disease type 3 clinical study, as well as our overall Gaucher clinical development strategy." | ||||
| Over a quarter ago | ||||
Avrobio announced that… Avrobio announced that the FDA has granted Rare Pediatric Disease Designation to AVR-RD-02, a first-in-class investigational gene therapy that genetically modifies patients' own hematopoietic stem cells, or HSCs, to treat Gaucher disease, a rare lysosomal disorder that can lead to multiorgan pathology, clinical morbidity and early mortality. | ||||
Avrobio announced that… Avrobio announced that the sixth and final patient has been dosed in the collaborator-sponsored, Phase 1/2 clinical trial of an investigational gene therapy for the treatment of cystinosis. Cystinosis is a life-threatening disease that causes progressive multi-organ damage, including early, acute kidney disease progressing to end-stage kidney disease. | ||||
