Latest news with #MarcDunoyer
Newsweek
16-05-2025
- Business
- Newsweek
The New Era of Life Sciences: The Future is Now
When we published our 2024 report, children suffering from Leber Congenital Amaurosis 4 (LCA4)—a severe inherited retinal dystrophy—had only hope that one day they might see the world. Today, thanks to an investigational gene therapy, we know that the 11 children who participated in the trials can, for the first time in their lives, discern a toy, recognize a human face and even spot a grain of sand. The biotech behind this achievement is MeiraGTx, which has now filed its therapy for approval under exceptional circumstances in the U.K., which would expedite the process. 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. Click here to download the full report
The Guardian
17-03-2025
- Business
- The Guardian
AstraZeneca pays up to $1bn for biotech firm ‘that could transform cell therapy'
AstraZeneca has struck a $1bn (£773m) deal to buy a Belgian biotech company that specialises in cancer immunotherapies, the latest in a string of acquisitions that also yielded positive results for a late-stage rare disease drug on Monday. EsoBiotec, a small privately held firm founded four years ago in Mont-Saint-Guibert, develops in-vivo cell therapies that empower the immune system to attack cancers, and could offer many more patients access to cell therapy treatments provided in minutes rather than weeks. The deal marks the latest acquisition by Astra, which is Britain's biggest listed company valued at £186bn, as the pharmaceutical group continues to expand its global operations. EsoBiotec's treatment uses highly targeted lentiviruses to deliver genetic instructions to specific immune cells, such as T-cells, which program them to recognise and destroy tumour cells for cancer treatment, or tackle autoreactive cells (that target the body's own tissues or cells) for potential use in autoimmune diseases. This means cell therapies can be administered through a simple injection, and EsoBiotec describes them as 'cost-effective, off-the-shelf therapies'. In traditional cell therapies, cells are taken from a patient, genetically modified outside the body, and then put back into the patient as a medicine after immune cell depletion, which usually takes weeks. Susan Galbraith, the executive vice-president of oncology research and development at AstraZeneca, said: 'We believe it has the potential to transform cell therapy and will enable us to scale these innovative treatments so that many more patients around the world can access them. 'EsoBiotec will accelerate and expand the impact of our recent investments and marks a major step forward in realising our ambition to harness the full potential of cell therapy.' EsoBiotec recently launched a clinical trial for multiple myeloma, a type of bone marrow cancer. AstraZeneca, which fended off a hostile £69bn takeover approach from the US group Pfizer in 2014, will pay $425m initially for the Belgian company, and up to $575m based on development and regulatory milestones. It has been beefing up its drug portfolio with targeted acquisitions, such as China's Gracell Biotechnologies, which specialises in cancer therapy known as CAR-T that modifies a patient's cells to fight the disease. AstraZeneca's biggest deal was the $39bn acquisition of the US rare disease drug developer Alexion in 2020, but it was criticised for paying a high price. Marc Dunoyer, who runs the division and is AstraZeneca's chief strategy officer, defended the deal as a 'fantastic acquisition' last month when the company took a $753m hit for scrapping one of the Alexion drugs, while two other products from the acquisition have also been abandoned. Separately, Britain's biggest pharmaceutical group said on Monday that its Imfinzi treatment had been approved in the EU as the first and only immunotherapy for patients with limited-stage small cell lung cancer, whose disease has not progressed after platinum-based chemoradiation therapy. Sign up to Business Today Get set for the working day – we'll point you to all the business news and analysis you need every morning after newsletter promotion AstraZeneca also announced that a drug for a rare endocrine disease, hypoparathyroidism, which can lead to bone density loss and osteoporosis, had met its goal of normalising calcium levels in the blood after 24 weeks. It was well tolerated by patients, and the trial continues as planned to 52 weeks. The company acquired the medicine along with the French rare disease specialist Amolyt Pharma in a deal worth more than $1bn a year ago. AstraZeneca also announced a licence agreement with South Korea's Alteogen to acquire the worldwide rights to its platform, to develop and commercialise subcutaneous formulations of several oncology medicines. Injecting medicines into the fatty tissue just under the skin can offer many advantages, including time savings for patients, clinical staff and health systems. AstraZeneca's share price reaction was muted, dipping by 0.3%.
