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The Companies Making a Difference for Rare Diseases: Meet the 2022 Black Pearl Awardees

Janeiro 2022

The Companies Making a Difference for Rare Diseases: Meet the 2022 Black Pearl Awardees

The EURORDIS Black Pearl Awards ceremony was created by EURORDIS to shine a light on the remarkable efforts to build a brighter future for everyone living with a rare disease. In the ceremony we award not just individuals but companies and the teams of people behind them.

In this article, you will meet this year’s recipients of the Company Awards, the European Alliance for Newborn Screening in Spinal Muscular Atrophy, Aparito and Lysogene.

So, who are this year’s awardees?

This year’s recipient of the Company Award for Innovation is Lysogene, whose activities are advancing the development of rare disease research and medicines. Lysogene’s founder, Karen Aiach, is a mother, patient advocate and entrepreneur determined to change the landscape for rare diseases affecting the central nervous system (CNS). When faced by her own child’s diagnosis with Sanfillipo (MPS III), she decided to use her business background to pull together a multidisciplinary team of experts to launch one of the first biotechs in the world developing gene therapies to treat paediatric central nervous disorders, and in her mission she has established a successful company constantly fighting for advances in rare diseases.

This year’s ​​Company Award for Patient Engagement recipient is The European Alliance for Newborn Screening in Spinal Muscular Atrophy (SMA). With an overarching goal of quicker and earlier diagnosis, the Alliance demands that by 2025, newborn screening programmes in all European countries include a test for spinal muscular atrophy for all newborn babies. The Alliance is a collaborative effort of many stakeholders all united around the goal of ensuring that newborn screening programmes in all European countries diagnose all newborn children with SMA.

Last but not least, the 2022 Company Award for Health Technology goes to Aparito! It’s founder, Elin Haf Davies was determined to help people with rare diseases get access to treatments to improve the quality of their lives. Aparito develops remote patient monitoring solutions to improve clinical trial outcomes, and combines the potential of innovative digital technologies with regulatory and clinical expertise to report patients’ needs and experiences accurately, and to improve healthcare for the rare disease community.

Why have they been chosen to be awarded?

The EURORDIS Company Award for Innovation is awarded to Lysogene to recognise them as a company which has established itself among major players driving European-led innovation. The award is also to recognise Lysogene’s advances towards delivering novel treatments in neuro degenerative and neuro developmental disease areas with high unmet medical needs. Furthermore, as one of the first biotechs in the world to create a position dedicated to liaising with families and patient organisations, this demonstrates the company’s continued readiness to engage with patients and caretakers as equal decision makers in the product development life cycle which also deserves recognition from this award. EURORDIS also wishes to celebrate the exceptional story of the company founder, as a mother, patient advocate and entrepreneur determined to change the landscape for severe rare diseases with unmet medical needs.

The European Alliance for Newborn Screening in SMA has been chosen to receive the Company Award for Patient Engagement to recognise its efforts to successfully and quickly create awareness and momentum towards an accelerated implementation of newborn screening for SMA all across Europe. The Alliance has put newborn screening in SMA on the political agenda at a country and European level, they have leveraged MEPs from different political groups to raise awareness on the topic and put pressure on the European Commission to set clear guidelines on newborn screening. The Award also recognises how the Alliance has created a map to follow for other initiatives across other rare diseases, and the award shines a light on the companies’ mutual engagement with patient advocates and multiple stakeholders around a single cause, which further reflects their commitment for and solidarity with the rare disease community.

The EURORDIS Company Award for Health Technology recognises Aparito as a company at the forefront of innovative technologies that report patient experiences accurately. By placing patient needs at the heart of the solution, Aparito’s expertise facilitates medicines development and contributes to addressing complex regulatory challenges. The award also recognises Aparito’s collaborative approach, working across rare diseases and alongside patient organisations, clinicians and sponsors. With the ever-increasing pace of developments in science and technology, Aparito’s remarkable journey in the field of remote patient monitoring is testament to the potential of digital solutions to profoundly impact the delivery of healthcare for people living with a rare disease.

A huge congratulations to the European Alliance for Newborn Screening in SMA, Lysogene and Aparito, and the incredibly dedicated people behind these companies and initiatives, for their outstanding efforts and innovation in the field of rare diseases.

Want to see their representatives receive their awards, and much more? Register now to watch the Black Pearl Awards for free, taking place online on Tuesday 8th February 2022 at 18.00 CET!


By Clara Maddison, EURORDIS Digital Communications Intern