Philanthropic drug development: understanding its importance, mechanisms, and future prospects.

Philanthropic drug development (PDD) addresses gaps in traditional pharmaceutical innovation, particularly for rare and underserved diseases. Cost and timeline challenges discourage new investments, especially in niche therapeutic areas. Patient organizations (POs) are uniquely positioned to help to reduce development challenges by providing expertise, supporting early research, fostering collaborations, and driving patient-centered clinical trials.

Assessing vaccine hesitancy and vaccine literacy among the European prison population and staff: A multicentre observational study.

Vaccination is the most efficient and cost-effective public health intervention. Prison population, for its low social distancing, constant turnover, and high percentage of migrants, should be an important target of vaccination campaign. However, vaccination coverage in prison is low.

Implementation of COVID-19 vaccination services in prison in six European countries: translating emergency intervention into routine life-course vaccination.

Background: Evidence has shown that the risk of transmission of SARS-CoV-2 is much higher in prisons than in the community. The release of the COVID-19 vaccine and the recommendation by WHO to include prisons among priority settings have led to the inclusion of prisons in national COVID-19 vaccination strategies. Evidence on prison health and healthcare services provision is limited and often focuses on a single country or institution due to the multiple challenges of conducting research in prison settings.

Editing via RNA -Splicing in RDEB-Derived Skin Equivalents.

Mutations in the gene lead to malfunction, reduction or complete absence of type VII collagen (C7) in the skin's basement membrane zone (BMZ), impairing skin integrity. In epidermolysis bullosa (EB), more than 800 mutations in have been reported, leading to the dystrophic form of EB (DEB), a severe and rare skin blistering disease associated with a high risk of developing an aggressive form of squamous cell carcinoma. Here, we leveraged a previously described 3'-RTMS6m repair molecule to develop a non-viral, non-invasive and efficient RNA therapy to correct mutations within via spliceosome-mediated RNA -splicing (SMaRT).

5'RNA -Splicing Repair of Mutant Transcripts in Epidermolysis Bullosa.

Mutations within the gene underlie the inherited recessive subtype of the blistering skin disease dystrophic epidermolysis bullosa (RDEB). Although gene replacement approaches for genodermatoses are clinically advanced, their implementation for RDEB is challenging and requires endogenous regulation of transgene expression. Thus, we are using spliceosome-mediated RNA -splicing (SMaRT) to repair mutations in at the mRNA level.