Immusoft Granted Orphan Drug Designation for Treatment of Rare Childhood Disease

May 2, 2018 |

3 min read

Proprietary system to be used to reprogram patients’ cells to produce treatment

SEATTLE--(BUSINESS WIRE)-- Immusoft Corporation, a Seattle, Wash.-based gene therapy company, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to treat the first indication in the company’s pipeline, MPS I (Mucopolysaccharidosis type I).

MPS I is a rare, childhood genetic disease that affects the body’s ability to produce an essential enzyme to break down long sugar chains inside cells. When the sugar chains can’t be broken down and disposed of, they accumulate in the body’s cells, which causes progressive damage.

Conventional treatment for MPS I involves injecting a manufactured replacement enzyme to break down the material in the cells. The enzyme breaks down quickly, however, and children with the condition must go through regular and consistent injections. The process is both time consuming and expensive.

Immusoft seeks to dramatically improve the treatment of MPS I through the use of its Immune System Programming (ISP™) system. ISP involves reprogramming a patient’s B cells, a type of immune cell, outside the body to produce therapeutic proteins. The technology originated in a B cell culture system invented by Nobel laureate David Baltimore, Ph.D., and was licensed and extensively improved upon by Immusoft.

The FDA’s Orphan Drug Designation offers incentives to companies to accelerate treatments for rare diseases such as MPS I. It is an important step for Immusoft’s first planned clinical application.

“We are pleased to have received FDA Orphan Drug Designation to treat MPS I using our proprietary culture system called Immune System Programming,” said Sean Ainsworth, Immusoft’s CEO and Chairman of the Board. “This is an important designation that will allow us to move forward more rapidly with the hope of treating and improving the lives of children with this condition.”

Severe MPS I occurs in about 1 in 100,000 births and symptoms appear within a child’s first year of life. In what is referred to as attenuated MPS I, symptoms appear later in childhood. It occurs in about 1 in 500,000 births.

ABOUT IMMUSOFT

Immusoft Corporation’s (immusoft.com) mission is to treat diseases using its breakthrough technology platform called Immune System Programming (ISP™). The technology instructs a patient’s cells to produce gene-encoded medicines (biologics). Cells that are reprogrammed using ISP become miniature drug factories that are expected to survive in patients for many years. Founded in 2009, Immusoft is based in Seattle, Wash.

FORWARD-LOOKING STATEMENTS

Statements in this press release that are not strictly historical are forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995. Forward-looking statements are based on management’s current plans, estimates and projections. The beliefs, assumptions and expectations on which these statements are based can change as a result of many possible events or factors, not all of which are known to Immusoft Corporation or are within its control. Immusoft Corporation undertakes no obligation to update any forward-looking statement in light of new information or future events. Actual results or outcomes may differ from those implied by the forward-looking statements as a result of a number of operational, scientific, regulatory and related risks and uncertainties.