Rare Beyond The Square
Global Genes is proud to bring you RARE Beyond the Square, in collaboration with the Biotech Showcase, to highlight rare disease progress and innovation, share information and facilitate partnering and networking among companies, investors and rare disease communities. All registered Biotech Showcase attendees will have access to RARE Beyond the Square company presentations and program sessions.
LIVE SESSION: Advancing patient-centered value frameworks for novel therapeutics in rare disease
While scientific advances have enabled the development of novel therapeutics to tackle genetic and rare diseases, healthcare infrastructure has struggled to keep pace in providing sustainable pathways to access for patients receiving them. Compounded with the high cost of development with limited markets, this has created an increasingly complex landscape for rare disease patients and companies looking to gain market access for their products. Hear about the current landscape for rare disease reimbursement access, where key challenges exist in developing sustainable pathways, and what types of new value frameworks and assessment models are being discussed to address them. This panel will take place as part of RARE Beyond the Square at Biotech Showcase hosted by Global Genes.
Thursday, 14 January 2021 8:30am - 9:30amPST/PDT (Pacific Daylight, GMT-7)
LIVE SESSION: Leveraging platform approaches to gene therapies to accelerate therapeutic development for monogenic diseases
While rare disease therapeutic development has accelerated in recent years and continues to be a major focus for biopharmaceutical companies and investors, 95%+ of rare diseases are still lacking an approved therapy or treatment. Hear how platform approaches to gene therapies and editing have the potential to enable a more scalable, and inherently patient-centric, approach to clinical development. This panel will take place as part of RARE Beyond the Square at Biotech Showcase hosted by Global Genes.
Wednesday, 13 January 2021 8:30am - 9:45amPST/PDT (Pacific Daylight, GMT-7)
OPEN SESSION: From de-risking to value creation – The evolving role of patient communities in rare disease drug development and commercialization
As patient communities and advocacy leaders become more deeply embedded in drug development, especially in rare diseases, it is imperative that organizations understand how patient engagement drives value beyond de-risking. Hear insights on how patients are changing the landscape for the development of novel therapeutics and how researchers, drug developers and funders can better leverage patient-centric approaches to optimize value throughout development. Delve into how patient advocacy engagement in drug development can act as a driver of investor value and learn how patient advocates are becoming market makers. This panel is open to the general public and will take place as part of RARE Beyond the Square at Biotech Showcase hosted by Global Genes.
On Demand PST/PDT (Pacific Daylight, GMT-7)
A film selection from The Disorder Channel: Unfixed - Covid: How We're Affected
The Unfixed series of films, as seen on The Disorder Channel, illustrates how COVID–19 has affected rare families from the lenses of Body, Mind, and Spirit. Biotech Showcase registrants will have special access to view the film “Unfixed - Covid: How We're Affected ” on-demand in early January 2021 and through the conclusion of the conference in partneringONE. The Unfixed series of films address Tourettes, Chrons, trigemial neuralgia, Mal de debarquement, ALS, Parkinsons, narcolepsy, fibromyalgia, TBI, synkinesis, transverse myelitis, polycythemia vera, asthma, and EDS. This film is included as part of RARE Beyond the Square program at Biotech Showcase hosted by Global Genes.