RARE Daily

Avrobio, Catalyst, Complete Offerings

February 19, 2020

Clinical-stage gene therapy developer Avrobio and rare bleeding disorders therapy developer Catalyst Biosciences competed public offerings.

Avrobio raised $100 million through an offering of 4.4 million shares at $23 a share.

Avrobio says it plans to use proceeds from the offering to fund its current programs in Fabry disease, cystinosis, Gaucher disease, and Pompe disease, among other things. In October 2019, the company dosed its first patient in a clinical trial of its experimental gene therapy program for cystinosis, a devastating lysosomal storage disease. Its lentiviral-based gene therapy is derived from a patient’s own hematopoietic stem cells, which are genetically modified to produce functional cystinosin, a crucial protein that patients with cystinosis lack.

Catalyst raised $34.5 million through an offering of 5.3 million shares at $6.50 a share.

The company is focused on systemic complement mediated disorders, with a clinical stage pipeline in hemophilia A and B that are designed to improve on current, intravenously delivered therapies for these disorders. Catalyst recently presented positive interim results from a mid-stage trial of its experimental therapy for hemophilia B that is designed to maintain levels of Factor IX above 12 percent in patients with severe hemophilia B.

In July 2019, Catalyst reported positive outcomes from a phase 2 trial of its lead compound marzeptacog alfa (activated) in patients with hemophilia A or B with inhibitors, which met the primary endpoint of reduction in annualized bleeding by more than 90 percent compared to pretreatment. 

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