Gene Therapy bringing new hope to patients and families

Top Story: Gene Therapy bringing new hope to patients and families

“Still tear up now thinking about it, just knowing that your daughter is not going to be able to see…” recalled by Ryan Troxel when he found out that his daughter, Molly Troxel, had an inherited genetic disease that led to her early childhood blindness.

At the time of the diagnosis, there was no cure for the condition. But thanks to the advances in gene therapy, Molly, now a 16-year-old junior, can now see many of the things that her family once had to describe to her. Effective treatments for some of the most challenging diseases known to medicine, gene therapies have offered hope to Molly and others.

Molly suffers from Leber congenital amaurosis (LCA), a debilitating condition responsible for congenital blindness. It is caused by genetic mutations in more than a dozen genes and affects roughly 3 in every 100,000 infants. In Molly’s case, she has unfortunately inherited from her parents a faulty copy of a critical vision gene known as RPE65. To target and restore the expression in RPE65, Spark Therapeutics has developed a gene therapy known as Luxturna, which uses genetic materials such as DNA to manipulate a patient’s cells for treatment. Specifically, this process involves adding engineered genes to a patient’s cells to replace inactivate, missing, or malfunctioning genes.

Dr. Daniel Chung, Clinical Ophthalmic Lead, Spark Therapeutics will be speaking at the 2nd annual Ophthalmic Drugs conference this November, focusing on the ‘New horizon for treatment of inherited retinal dystrophy.’

Dr. Chung will discuss the first ever FDA-approved gene therapy, Voretigene, which has been created to treat a rare inherited retinal disease. He will also explore other up-coming gene therapy trials in the pipeline at Spark Therapeutics and the market access difficulties for gene therapy.

Download the 2018 agenda online to find out more and see the full speaker line-up, as well as the attendee list from last year at:

For those looking to attend, there is currently a £200 early-bird saving, ending September 28th.

SMi Presents the 2nd annual:
Ophthalmic Drugs 2018
Date: 26th – 27th November 2018
Workshops: 28th November 2018
Location: Copthorne Tara Hotel, London UK

Sponsors: Experimentica | Nemera


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For all media inquiries contact Pavan Solanki on Tel: +44 (0)20 7827 6048 / Email:

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