Please login to the form below

Reflections of a Fireside Chat from the World Orphan Drug Congress 2020

Hear from Trish Shepherd COO, Patient & Brand Communications, OPEN Health

From junior doctor to gene therapy CEO, how one person’s drive to make rare disease curable is shaping the future of medicine.

It was a truly touching story to hear Dr Gaspar recant his early days working with young, severely immunocompromised patients at Great Ormond Street. Dr Gaspar knew that most of his patients would die within their first year without any therapy. Although bone marrow transplantation was an option for some patients, it was not without risk of complications.


Dr Gaspar was first told of a single patient candidate for gene therapy while doing his rounds in 1993. He was initially convinced it was a Senior Doctor prank on a trainee, but as the years passed Dr. Gaspar saw that one patient return to the hospital with a fully healthy immune system and he was inspired to return to the lab to explore. And he did. In 2001, he went on to test the innovations he and his colleague were exploring in human clinical trials. The success these approaches received garnered much interest: first beyond the academic setting and then across regions and continents. The next step was clear - a commercial entity was needed to deliver more solutions to more people.

The result was Orchard Therapeutics, a company dedicated to setting up a process that enables the treatment cells to travel so that patients, who are already living with debilitating diseases, do not have to. As a business, Orchard Therapeutics want to introduce as many therapies as possible and, in order to enable a broader reach, are eager to work with regulators to explore reimbursement models from single payments, to annualised payments or payment by results.


When asked what it is like as a clinician to see this level of success, Dr Gaspar replies that there is nothing more rewarding than treating a patient with a therapy that you have created – it is, truly, an unbeatable moment.

Dr Gaspar has now moved on from his clinical duties to focus fully on his CEO responsibilities at Orchard Therapeutics, but remains certain that the options for clinicians when faced with rare genetic disease diagnoses, such as Severe Combined Immune Deficiency (SKID) and Metachromatic leukodystrophy (MLD), are different. The conversations with the families of patients will be entirely more positive in light of the new available options. The face of the disease is, thanks to Dr Gaspar and his colleagues’ work, now permanently changing for the better.

From this fireside chat, you can see the desire to introduce more curative therapies still burns bright.

Pink divider

Here at OPEN Health Patient & Brand Communications we pride ourselves on our deep understanding of the patient experience in rare disease and have significant expertise in consulting on the delivery of innovative treatments in complex treatment pathways, as well as providing niche patient support platforms to help patients and their families better understand novel treatments.

If you’d like to learn more, we’d love to hear from you and look forward to working together to help introduce more novel therapies to the people that need them most.

Trish Shepherd
COO, Patient & Brand Communications

+44 (0) 7387 253317

9th September 2020


Company Details

OPEN Health

+44 1628 481112

Contact Website

The Weighbridge
Brewery Courtyard
High Street
United Kingdom

Latest content on this profile

Announcing OPEN Health New Communications CEO, Margot Hannah
OPEN Health is pleased to announce the appointment of Margot Hannah as the new Chief Executive Officer of OPEN Health Communications
OPEN Health
OPEN Health Acquires ARK
OPEN Health has today announced the acquisition of London-based agency ARK to further strengthen omnichannel capabilities
OPEN Health
Rare thoughts & outcomes - navigating pathways to better outcomes in rare

OPEN Health
How to pick the perfect training program for your healthcare professionals
You know your team needs training. But not all training providers and programs are created equal. Last week, we shared the benefits of working with specialist learning and development (L&D) teams to maximize your training investment. Once you’ve identified some potential collaborators, it’s time to take a closer look at what should be in the packages your training provider offers. Here are six vital features you should prioritize to select the training that will help your team fulfil their potential as partners in delivering high-quality and effective healthcare to patients.
OPEN Health
Maximize your training investments with learning and development specialists
Medical affairs teams must train regularly to stay up to date with existing and emerging therapy areas, products, and devices. But finding time to train is tough, so what can you do to ensure your training programs are as cost-effective, time-efficient, and high quality as possible?
OPEN Health
A day in the life of a PR Account Director
We asked Laura Glover to give us an insight into her role as a PR Account Director at OPEN Health
OPEN Health