Orchard RX has filed an amended F-1 form with the U.S. Securities and Exchange Commission (SEC) for its initial public offering (IPO). The company is offering 13.33 million American depositary shares (ADSs) for $14 to $16 apiece, with an overallotment option for an additional 2.0 million ADSs. Each ADS represents one ordinary share. At the maximum price, the entire offering is valued up to $245.33 million. The company intends to list its ADSs on the Nasdaq under the symbol ORTX.

The underwriters for the offering are JPMorgan, Goldman Sachs, Cowen and a Wedbush PacGrow.

This is a commercial-stage, fully integrated biopharmaceutical company working to treat patients with serious and life-threatening rare diseases through autologous ex vivo gene therapies. The gene therapy approach seeks to transform a patient’s own, or autologous, hematopoietic stem cells into a gene-modified drug product to treat the patient’s disease through a single administration.

The company achieves this outcome by utilizing a lentiviral vector to introduce a functional copy of a missing or faulty gene into the patient’s autologous hematopoietic stem cells through an ex vivo process, resulting in a drug product that can then be reintroduced into the patient at the bedside.

Orchard intends to bring potentially transformative therapies to the broadest number of patients suffering from rare diseases. The indications that the firm is initially targeting in its primary immune deficiencies and neurometabolic franchises alone have a combined annual incidence rate of between 1,000 and 2,000 patients in markets around the world where treatments for rare diseases are often reimbursed.

Based on this, management believes the total addressable market potential in the diseases areas underlying its five lead programs could be greater than $2 billion annually. In addition, certain indications such as X-linked chronic granulomatous disease and Wiskott-Aldrich syndrome have large existing populations with pre-existing disease that could be eligible for these treatments upon receiving marketing approval, which could increase the size of its market opportunity further.

The company intends to use the net proceeds from this offering to fund the ongoing development of its product candidates, with the remainder going toward working capital and general corporate purposes.

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