AVROBIO, a clinical-stage biotechnological company developing transformative, life-changing gene therapies for rare diseases, today announced the completion of a $60 million Series B funding Funding will be used to advance multiple gene therapies from AVROBIO’s proprietary lentiviral platform, including the company’s lead gene therapy, AVR-RD-01, currently in phase 1 for Fabry disease, as well as three additional gene therapies for other lysosomal storage disorders, Gaucher disease, cystinosis and Pompe disease. The Series B round was co-led by Cormorant Asset Management and Surveyor Capital (Citadel Company) and included the participation of Aisling, Brace Pharma Capital, Eventide Asset Management and Morningside, together with existing investors Atlas Venture, SV Health Investors and Clarus Ventures.
AVROBIO plans to launch a phase 2 clinical trial this year with AVR-RD-01 in Fabry Disease on the basis of promising initial six-month clinical results in the ongoing phase 1 trial. In the first patient with Fabry disease in the Phase 1 trial, treatment with a single dose of AVR-RD-01 resulted in normal plasma activity of the enzyme Î±-galactosidase A, a genetically deficient lysosomal enzyme in patients with Fabry disease. In addition, the company plans to initiate the clinical development of two additional candidates for gene therapy for cystinosis and Gaucher disease by mid-2019.
“Gen therapy is a class of medicines that have made breakthroughs in the biotechnological landscape, and AVROBIO has established a strong position with its lentiviral platform focused on significant therapeutic and market opportunities for gene therapy for lysosomal storage diseases,” said Bruce Booth, D. Phil, Phil Partner at Atlas Venture and Chairman of the Board of Directors of AVROBIO “AVROBIO has made remarkable progress with its gene therapy platform, including a very compelling initial clinical response in the first Fabry disease patient to receive the company’s lead gene therapy candidate. We have great confidence in the high-quality management team and the rigorous science and clinical programs behind gene therapy for Fabry disease and a number of other lysosomal storage disorders. “That’s right.
With AVR-RD-01 and its line of gene therapy candidates, AVROBIO is the first company to move into the lysosomal storage gene therapy clinic, a class of rare genetic diseases in which patients have a defective gene that produces an enzyme or protein that serves a vital metabolic function. Initially, the company applied its proprietary lentiviral gene therapy platform to lysosomal storage disorders and will also apply the platform to a wide range of other diseases where the systemic delivery of gene therapy may be therapeutically beneficial. Lentiviral vector technology is a gene transfer system for the stable addition of genes to the patient’s own cells, designed to provide permanent genomic integration for long-term and potentially life-long curative benefit to patients.
“We are pleased with this exceptional group of investors supporting our plans to build AVROBIO as a leader in the field of lentiviral gene therapy,” said Geoff MacKay, President and CEO of AVROBIO. “We are excited to continue to rapidly advance our clinical program for AVR-RD-01 in Fabry Disease, while also moving two additional gene therapies from our pipeline to clinical trials. We are at the forefront of applying gene therapy as a potential single-dose curative treatment for lysosomal storage disorders, and our vision is to use our lentiviral platform to have a broader impact on patients by shifting the paradigm to a wide range of other diseases. “That’s right.
Leerink Partners acted as sole financial advisor to AVROBIO’s Series B financing
About AVR-RD-01 and the initial clinical outcomes
AVR-RD-01 is a lentiviral gene therapy that is being studied as a single-dose therapy with a lasting and potentially life-long healing benefit for patients with Fabry disease. AVR-RD-01 uses a state-of-the-art lentiviral vector system that is an efficient and proven gene transfer system for the stable addition of genes to the patient’s stem cells. In patients with Fabry disease, their CD34+ stem cells are extracted and isolated, and the cells are transduced by a lentiviral vector that carries a normal GLA gene to generate AVR-RD-01 gene therapy. AVR-RD-01 is then infused back into the patient on an outpatient basis with the aim of restoring normal GLA gene expression so that the enzyme Î±-galactosidase A is produced by the patient’s own body.
In October 2017, AVROBIO presented initial six-month clinical data for the first patient with Fabry disease treated with AVR-RD-01. The single dose of AVR-RD-01 allowed the patient to achieve and maintain normal plasma activity of Î±-galactosidase A, a genetically deficient lysosomal enzyme in patients with Fabry disease, at six months.
AVROBIO’s Lentiviral Gene Therapy Platform
AVROBIO has designed a state-of-the-art, 3rd generation, 4-plasmid lentiviral vector platform, an efficient and proven gene transfer system for the stable addition of genes to the patient’s own CD34+ stem cells. Lentiviral vector technology is designed to provide continuous genomic integration and can be applied to a range of diseases where systemic delivery may be therapeutically beneficial. AVROBIO’s platform includes patented technology, unique manufacturing processes, expertise in gene therapy and proprietary tools.
AVROBY, Inc. , a leader in lentiviral-based gene therapy, is a clinical-stage company developing disruptive therapies that have the potential to transform patients’ lives at a single dose. The company focuses on the development of its gene therapy candidate, AVR-RD-01, in Fabry disease, as well as additional gene therapy programs in other lysosomal storage disorders, including Gaucher disease, cystinosis and Pompe disease. AVROBIO’s lentiviral platform has a wide potential for other rare and non-rare genetic diseases. AVROBIO is headquartered in Cambridge, MA and has offices in Toronto, Ont. For more information, please visit www Avrobio’s Come on,
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