3 min read

UBC a Finalist for Clinical Research Team of the Year

The Citeline Awards by Informa Pharma Intelligence have named UBC’s Gene Therapy Registry Team as a shortlisted finalist for the Clinical Research Team of the Year category.
The Citeline Awards by Informa Pharma Intelligence named UBC's Gene Therapy Registry Team as a shortlisted finalist for the Clinical Research Team of the Year category.

The UBC Gene Therapy Registry Team undertook a highly successful collaboration in the conduct of a prospective observational registry of patients with Spinal Muscular Atrophy (SMA). The program successfully enrolled approximately 300 patients into the global registry within an initial two-year period, which produced robust data used to meet regulatory objectives, inform SMA experts, and support publications. This data has ultimately contributed to more thoroughly understanding the efficacy and safety of advance therapies for SMA.

SMA is a neurogenetic disease affecting the central nervous system, peripheral nervous system, and voluntary muscle movement (skeletal muscle). SMA most often affects babies and children and makes it hard for them to use their muscles, and children can have trouble controlling head movement, sitting without help, and even walking. In some cases, they can have trouble swallowing and breathing as the disease gets worse. The main objective of this registry is to assess the long-term outcomes of patients with a diagnosis of SMA in the context of advances in treatment options.

SMA used to be one of the most common genetic causes of infant mortality. New disease-modifying treatments have changed the disease trajectories and the best results are seen if treatment is initiated in the pre-symptomatic phase of the disease. Systematic real-world data collection and additional clinical trials are needed to fill evidence gaps concerning safety and efficacy. The data that has and will be gathered in this registry is ambitiously addressing evidence needs for the SMA community at large.

The Gene Therapy Registry Team is a cross-functional team that has worked daily for over two years to propel the registry forward. This includes work in engaging the SMA community, effectively onboarding global institutions, driving data-sharing partnerships, and disseminating the knowledge gained from the data.

UBC is proud of the work the Gene Therapy Registry Team has performed in combatting such a debilitating disease. This well-earned recognition highlights the important efforts that go on, often unnoticed, to effect change against rare diseases. To learn more about how we can generate evidence for the safety and efficacy of your therapy.

Other Recent Posts

Press
5 min read

UBC Successfully Implemented REMS Administration Software DuringHL7® FHIR® Connectathon

UBC demonstrated our commitment to optimizing safe medication use and health outcomes by participating in the HL7® FHIR® Connectathon.
Press
5 min read

UBC Selects Oracle Cloud Safety Technology for its Safety Programs

UBC selected Oracle Life Sciences' safety case management solution.
Press
2 min read

UBC Receives CRO Leadership Award Again

UBC was honored as a winner in the 2023 CRO Leadership Awards by both big and small pharma in the Reliability category.

Thank You for Connecting with UBC

Lorem ipsum dolor sit amet consectetur. Mi proin enim ipsum nascetur. Venenatis odio scelerisque non netus ac et pellentesque.

What You Can Expect Next

Lorem ipsum dolor sit amet consectetur. Cras aliquam erat eget magna sollicitudin.

Get Ready to Change Your Business

Lorem ipsum dolor sit amet consectetur. Mi proin enim ipsum nascetur. Venenatis odio scelerisque non netus ac et pellentesque.

Service Request

Bekki Bracken Brown

President & Chief Executive Officer

Bekki Bracken Brown serves as the President and CEO of UBC, guiding the company’s mission and values, including the improvement of access for patients to receive better outcomes. She oversees all aspects of UBC, such as operations, business growth strategy, sales and marketing, and acquisition support.

With over 20 years of industry experience, Ms. Brown brings knowledge from a successful career in senior management from her tenure at Quintiles, INC Research, and, most recently, with Syneos Health. She’s been a member of the North Carolina BIO Board of Directors since 2019. She is also a member of the Healthcare Businesswomen’s Association — Southeast Chapter and CHIEF, an organization that supports women executive leaders. Ms. Brown earned her bachelor’s degree at Duke University.