Genetic Diseases:?It’s a disease that often strikes people in the prime of life, slowly destroying control over body, mind and personality. It inevitably kills the patient, but usually only after at least a decade of suffering. The underlying cause of Huntington’s disease – a dominant mutation on Chromosome IV – was discovered almost 25 years ago. Now the first trials to target the condition at its source have begun. Chances of finding a cure have never been more realistic.
ADVERTISEMENT
AYOXXA’s LUNARIS platform:?In a case study "Translational proteomics: a new perspective for
ophthalmology research, Ayoxxa gives insights into the capabilities of its Lunaris multiplex protein
analysis platform.
Progress in process engineering and media optimisation has resulted in significant productivity increases in upstream processing driving transformations at drug developers and CDMOs. At BPI Europe in Amsterdam, companies showed strategies and product launches pinpointing the way towards what could become a new industry paradigm: continuous processing and flexible modular production plants, which both address changed production needs.
The possibilities offered by CRISPR/Cas9, TALENs and other new genome editing technologies are making the mouths of plant breeders water – and not just because the methods give seed developers new options when it comes to optimising traits such as yield, resistance to environmental stress, diseases or pests. Gene-edited plants could soon make inroads even with Europe’s strict regulators, and throw open the door to tastier, healthier food.
Belgian, Swiss, and US researchers have found a new way to boost efficacy in cancer immunotherapy. In mid-April, they reported that cancer cells actively block tumour infiltration of cytotoxic T cells, but that this process can be reversed using angiogenesis blockers. Some Big pharma companies have already jumped on the bandwagon of angiogenesis/PDL1 combination therapy.
French green chemistry pioneer, Carbios SA (Clermont-Ferrand) announced in March that it has secured equity financing of €2.5m from Kepler Cheuvreux through the acquisition of 380,000 shares over 24 months.
Lab equipment giant Thermo Fisher Scientific Inc. is growing even bigger.
Since US senators in February conveyed the impression that drug developers might have misused market monopolies related to orphan drugs (ODs) to overprice their compounds, the status has come under closer scrutiny. Neither the US nor Europe currently have consistent approaches for deriving value for OD therapies. Pooling expertise and scattered patient data across borders could help ease both diagnosis and development, providing a foundation for pricing and giving patients much faster access to treatments.
The concept has been around for more than a century, but recent advances in the field are causing numerous experts to say it’s widely underused. The goal of allergen immunotherapy (AIT) is to induce a specific immune tolerance by confronting patients with increasing levels of allergens. AIT developers are now working on improving efficacy and safety, while at the same time trying to speed up the still lengthy process of desensitisation. Peanuts in particular are in the crosshairs, as they cause one of the most life-threatening common allergies in humans.
Uncertainty regarding the amount of future funding and political support from the European Commission for the bioeconomy is set to hamper investments into the switch from oil-based industry production to renewable, bio-based manufacture in Europe. The schedule for the review of the bioeconomy strategy has been delayed until the end of 2017. More importantly, it is not yet certain if the strategy will be updated.