SMART CRC partners with EB Research Partnerships Australia on a 10-year plan to accelerate therapies for Epidermolysis Bullosa
EB Research Partnerships Australia (EBRPA), in collaboration with the SMART CRC, is working to accelerate the development of therapies that ease the burden of Epidermolysis Bullosa (EB).
EB is a rare and severe genetic condition caused by mutations in genes responsible for structural proteins that hold the skin together. The result is extreme skin fragility, chronic wounds, and in severe cases, life-limiting complications including infection, nutritional decline, and elevated cancer risk
Together, we are working to develop and manufacture therapies to help the approximately 500,000 people with this rare disease across the world, manage the debilitating symptoms of itch, pain and inflammation.
The challenge
While global efforts are progressing towards a cure for EB, Australia faces barriers around access, availability and manufacturing of therapies that could improve quality of life for patients today.
EBRPA Chair Nathan Burmeister who is father to Jacob, a 4-year-old living with Recessive Dystrophic EB, said he was hopeful that within the next decade, Australia would move closer to easing the burden of this life-threatening disease.
“Within 10 years, there is no doubt EBRP and the SMART CRC will develop at least one or two therapies together,” he said.
“Therapies that will ease the burden for patients while international efforts are focussed on finding a cure.”
The response needed
EBRPA’s focus is on outcomes and prioritising practical action and impact for patients.
“We want to work with people, industry and research organisations that want to put therapies in patients. Research is important, but we want and need practical action now.”
Many of the challenges facing EB, including limited drug access, difficulties repurposing therapies and manufacturing constraints, are systemic barriers shared across the rare disease sector.
Why partner with SMART CRC?
SMART CRC addresses the challenges EBRPA is working to overcome by strengthening Australia’s capability to manufacture, translate and deliver advanced and regenerative therapies.
“Many in the EB community have lived experience of all the problems the CRC is trying to solve by improving manufacturing of regenerative therapies. I have experienced drugs not being available in Australia, drugs not being able to be repurposed and the flow on effects of manufacturing issues. The impact that has on my son and our family is enormous.” Nathan said.
“The SMART CRC is a vehicle for EBRPA to align our needs with a nationally coordinated framework that will ultimately accelerate impacts and solve the challenges patients and their families are facing.”
Why itch? A patient-led research priority
EBRPA’s initial research focus within the SMART CRC is chronic itch- identified as the number one unmet need through the largest global EB research priority-setting initiative, involving more than 800 patients, carers and clinicians.
Chronic itch in EB is constant, sleep-disrupting, and largely unaddressed by existing therapies. It also plays a critical role in the “itch–scratch” cycle, driving further skin damage and complicating treatment outcomes.
The outcome and impact
While EBRP and SMART CRC are working collaboratively to deliver therapies for people living with EB, this partnership supports a stronger pathway for rare disease therapies in Australia. Partnerships involving industry, government, research and clinicians are critical for a strategic, sector wide effort.
“If we use EB as a model, we know that can and will benefit the wider community, the more than 2 million people living with a rare disease in Australia,” he said.