Korea With the bioPROTAC platform, we can, in principle, degrade essentially any problematic protein Jaehyun Choi, Co-CEO of Genexine, outlines the company’s transition from its established hyFc platform towards bioPROTAC, a novel protein degradation technology. He discusses GX-BP1, partnerships, regulatory challenges in Asia, and Genexine’s strategy of validating the platform through…
USA Pamela Gavin, CEO of the National Organization for Rare Disorders, discusses NORD’s origins in the advocacy movement that led to the Orphan Drug Act and its current mission to improve diagnosis, care, research, and policy for rare disease patients. She highlights the growing role of patient-led research, the need for…
USA Daniel Camardo is the President of Immedica Pharma US Inc. (Immedica), the US affiliate/subsidiary of Immedica Pharma AB, a Sweden-based rare disease company that entered the American market in 2024. Bringing together his experience building Astellas’s US presence and leading rare disease commercialization at Horizon, Camardo joined Immedica to lead…
Ireland The first thing I’d say to innovators is to ask the patients. Patient groups are ready to support innovation, make sure we’re measuring what matters, and delivering what’s meaningful Avril Daly brings a rare dual perspective to Europe’s rare disease and ophthalmology landscape: as CEO of Retina International, she represents…
MEA Once regarded as a strategic afterthought by international biopharma, the Middle East has undergone a profound transformation in recent years, emerging as an early adopter of next-generation therapies for rare diseases. Regional regulatory authorities are accelerating approval timelines, reflecting an aggressive institutional ambition to secure speedy access to cutting-edge medical…
MEA Long regarded as a clinical trials backwater, the Middle East has experienced a tremendous surge in clinical research over the past five years. This boom is fuelled by a global demand for representational equity and an urgent need to correct a stark imbalance, as Middle Eastern populations currently account for…
UAE It takes a great deal to become number one in a field, but the harder challenge is staying there Joe Henein, CEO of Dubai-headquartered NewBridge Pharmaceuticals, has built one of MENA’s most distinctive specialty pharma platforms over the past 15 years. With over 300 people across MENA, a pan-regional footprint…
Germany Recordati Rare Diseases aims to be a trusted partner in helping patients with rare diseases reach diagnosis and appropriate care sooner Rapid access to innovation alone does not solve the rare disease challenge in Germany. Despite being one of Europe’s most advanced pharmaceutical markets, patients still face long diagnostic journeys…
USA You cannot choose between performing and transforming. The organisations that succeed can do both As AI, shifting healthcare policy, growing pressure around affordability and access, and rapidly evolving models of commercialisation reshape the pharmaceutical industry, Deborah Glasser reflects on how Sanofi is positioning its specialty care business for the next…
USA Access works when all pieces are in place. Hearing directly from patients about what diagnosis or how a treatment impacts their lives is meaningful to everyone involved Scott Weintraub, Head of US Business at Alexion, AstraZeneca Rare Disease, discusses how his experience across commercial leadership, strategy, and rare disease innovation…
USA More than 40 years ago in 1983, the Orphan Drug Act was passed in the US to support and incentivise the development of rare disease therapies. Despite the over 6,000 orphan drug designations (ODD) granted by the FDA since then, it is estimated that only around five percent of rare…
GCC Healthcare transformation across the GCC is entering a new phase, driven by major investments in precision medicine, genomics, and healthcare system modernisation, with countries such as Saudi Arabia and the UAE positioning themselves at the forefront of global healthcare innovation. Against this backdrop, Bristol Myers Squibb is expanding its role…
See our Cookie Privacy Policy Here
