The future is now: Orchard’s potentially curative gene therapies

A wave of innovation is delivering breakthrough treatment options for rare inherited diseases that were once considered too challenging or impossible to treat

Orchard Therapeutics, a biotech with roots in the heart of London, is among leaders of a genetic revolution.

“When the human genome was mapped nearly 20 years ago, the notion that this genetic blueprint could advance therapies capable of finding and possibly fixing the genes responsible for some of the world’s most devastating diseases was an idea of the future,” says Bobby Gaspar, professor of paediatrics and immunology at the UCL Great Ormond Street Institute of Child Health.

Giovanni and Cecilia Price hold a photo of their sister, Liviana, who died at an early age from MLD, a diagnosis they share

Professor Gaspar, who has led multiple successful gene therapy clinical trials in immune deficiencies and other rare genetic diseases, is chief scientific officer at Orchard. A global entity with offices in London, Boston and the San Francisco Bay area, Orchard has the European-approved, commercial gene therapy product, Strimvelis®, together with six clinical-stage gene therapy products in development and a robust pre-clinical pipeline, all aimed at potentially curing a range of rare diseases with a single administration of gene therapy.

Orchard’s gene therapy approach is highly individualised and utilises gene-modified blood stem cells to target the underlying cause of disease. Blood stem cells are taken from the patient, genetically modified outside of the body by inserting a functional copy of a missing or faulty gene, and then transplanted back in the patient to potentially cure the disease through a single administration.

Mark Rothera, a seasoned biotech executive who has spent most of his career bringing treatments to patients with rare diseases, took the helm at Orchard in September 2017 after spending nearly 30 years in the industry and has grown the company from 35 employees to more than 200. Mr Rothera and his team took the company public last October and continue to add new programmes to the company’s growing portfolio, each of which leverages Orchard’s gene therapy approach.

“Everything I’ve launched previously has focused on slowing disease progression and trying to improve quality of life, but at Orchard we have the potential opportunity to take a child on a path to dying at five years of age, and with a single administration, provide them with a more normal life,” he says.

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Remembering Liviana

Amy and Brad Price’s daughter Liviana was born with a fatal genetic disorder called metachromatic leukodystrophy, or MLD, a fast-advancing disease of the brain for which no approved treatments exist.

Within days of Liviana’s diagnosis, the Prices’ other children were tested for MLD and the family learnt their younger son Giovanni also had the genetic defect responsible for this devasting condition.

The Prices heard about a clinical trial in Milan evaluating an investigational gene therapy to treat MLD in certain infants and young children. While Liviana’s disease was too advanced for her to participate in this trial, Giovanni was eligible.

He, and later his younger sister Cecilia, received the investigational treatment now referred to as OTL-200. Today, the children enjoy going to school, playing sports and finding ways to remember their sister.

“Although Livi is no longer physically here with us, she has left an important and lasting imprint on the tapestry of our family,” says Ms Price. “Our love for her endures and we keep her memory alive always.” 

This story represents a single patient experience in a clinical trial of OTL-200, an autologous gene therapy for the treatment of MLD. The safety and effectiveness of OTL-200 for MLD have not been established, and OTL-200 is not an approved therapy in any country.

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