The New Era of Life Sciences: The Future is Now
Stories like that of MeiraGTx illustrate the transformative power of gene therapies on patients. More broadly, they highlight the potential for rare diseases R&D to emerge as the principal driver of therapeutic innovation. "Rare diseases have become a critical area for exploring new treatment modalities because, in many cases, there is no existing therapy, or even a model. For patients who have no options, innovative approaches are important," shares Marc Dunoyer, the CEO of Alexion, the rare diseases subsidiary of AstraZeneca. Ironically, the very severity and uncommonness of these illnesses can also be a blessing, as it allows for bolder approaches by both researchers and regulators. Moreover, rare disease drugs have historically had higher approval success rates compared to other drugs. The Tufts Center for the Study of Drug Development found that orphan-designated drugs had a Phase-1-to-approval success rate of 17 percent, compared to 7.9 percent for non-orphan drugs. Benefiting from faster approval pathways and enhanced regulatory incentives, entrepreneurs in the field face better odds of innovating successfully.
And when they do, the implications of their therapies go far beyond their original target. "Many of the advances seen today, such as gene editing and RNA-based therapies, originated in rare disease research before moving into larger indications," says Dunoyer. Arcturus Therapeutics, for instance, is addressing cystic fibrosis via a new delivery mechanism. "Our cystic fibrosis program is a prime example, where the ability to inhale mRNA safely could have a huge impact on the pharmaceutical industry. The rare disease field offers a shorter regulatory pathway to approval and a higher likelihood of success. This makes it an attractive space for evaluating next-generation technologies like mRNA therapeutics," shares Arcturus' CEO, Joseph Payne, as the company is expecting results from their Phase 2 trials in 2025.
Matt Sause, CEO, Roche Diagnostics. Credit: Courtesy of Roche Diagnostics.
Matt Sause, CEO, Roche Diagnostics. Credit: Courtesy of Roche Diagnostics.
We can perform comprehensive genomic profiling to uncover the molecular basis of a tumor. This allows doctors to provide tailored treatments. Over time, this will enable us to move to a future where we understand the molecular drivers for cancer and can deliver truly personalized healthcare.
Jacob Thaysen, CEO, Illumina. Credit: Courtesy of Illumina.
Jacob Thaysen, CEO, Illumina. Credit: Courtesy of Illumina.
Whether it is providing clarity for families dealing with rare genetic disorders or enabling rapid and precise cancer diagnoses, our goal is to ensure patients receive the answers they need when they need them.
This report has been paid for by a third party. The views and opinions expressed are not those of Newsweek and are not an endorsement of the products, services or persons mentioned.
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Available from: Last accessed: April 2025 Roche Global Media RelationsPhone: +41 61 688 8888 / e-mail: Hans Trees, PhDPhone: +41 79 407 72 58 Sileia UrechPhone: +41 79 935 81 48 Nathalie AltermattPhone: +41 79 771 05 25 Lorena CorfasPhone: +41 79 568 24 95 Simon GoldsboroughPhone: +44 797 32 72 915 Karsten KleinePhone: +41 79 461 86 83 Kirti PandeyPhone: +49 172 6367262 Yvette PetillonPhone: +41 79 961 92 50 Dr Rebekka SchnellPhone: +41 79 205 27 03Roche Investor Relations Dr Bruno EschliPhone: +41 61 68-75284e-mail: Dr Sabine BorngräberPhone: +41 61 68-88027e-mail: Dr Birgit MasjostPhone: +41 61 68-84814e-mail: Investor Relations North America Loren KalmPhone: +1 650 225 3217e-mail: Attachment Media Investor Release Elecsys pTau181 CE Mark English
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The study showed the test was able to rule out Alzheimer's disease with a high negative predictive value (NPV) of 93.8% based on a 22.5% prevalence of amyloid positivity according to positron emission tomography (PET) scans, with 83.6% sensitivity. The rule out performance of the test was only minimally impacted by the patients' age, gender, body mass index or impaired kidney function. This global, prospectively-collected, diagnostic registrational clinical study was the first of its kind in the industry to investigate the test's clinical performance in a diverse patient population, aged 55-80 years old that reflects as closely as possible the patients who could benefit from the test. It involved a subset of patients from a wider study looking at a highly diverse set of patients with broad inclusion criteria, to ensure the test could be used effectively across different geographies and ethnicities. 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About Roche Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world's largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice. For over 125 years, sustainability has been an integral part of Roche's business. As a science-driven company, our greatest contribution to society is developing innovative medicines and diagnostics that help people live healthier lives. Roche is committed to the Science Based Targets initiative and the Sustainable Markets Initiative to achieve net zero by 2045. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit . All trademarks used or mentioned in this release are protected by law. References [1] Alzheimer's Disease International. World Alzheimer Report 2022 [Internet; cited April 2025]. Available from: . Last accessed: April 2025 Roche Global Media Relations Phone: +41 61 688 8888 / e-mail: [email protected] Hans Trees, PhD Phone: +41 79 407 72 58 Sileia Urech Phone: +41 79 935 81 48 Nathalie Altermatt Phone: +41 79 771 05 25 Lorena Corfas Phone: +41 79 568 24 95 Simon Goldsborough Phone: +44 797 32 72 915 Karsten Kleine Phone: +41 79 461 86 83 Kirti Pandey Phone: +49 172 6367262 Yvette Petillon Phone: +41 79 961 92 50 Dr Rebekka Schnell Phone: +41 79 205 27 03 Roche Investor Relations Investor Relations North America Loren KalmPhone: +1 650 225 3217 e-mail: [email protected] Attachment Media Investor Release Elecsys pTau181 CE Mark English Disclaimer: The above press release comes to you under an arrangement with GlobeNewswire. Business Upturn takes no editorial responsibility for the same. Ahmedabad Plane Crash
