Particulates in CGT guidance survey results - an ISCT process development & manufacturing committee perspective on cell and gene therapy industry knowledge awareness.

The International Society for Cell & Gene Therapy Particulates Working Group has surveyed the global industry on commonly used regulatory guidance documents pertaining to particulates across various cell and gene therapy (CGT) product-based chemistry and manufacturing controls. Responses were collated from academic and industrially based individuals involved in or making decisions for manufacturing, quality control and analytics. Two surveys covering therapy developers and manufacturing suppliers were created and distributed via e-mail, through focus groups and via social media platforms.

The Discovery and Preclinical Profile of ALG-000184, a Prodrug of the Potent Hepatitis B Virus Capsid Assembly Modulator ALG-001075.

Chronic hepatitis B (CHB) represents a significant unmet medical need with few options beyond lifelong treatment with nucleoside analogues, which rarely leads to a functional cure. Novel agents that reduce levels of HBV DNA, RNA and other viral antigens could lead to better treatment outcomes. The capsid assembly modulator (CAM) class of compounds represents an important modality for chronic suppression and to improve functional cure rates, either alone or in combination.

Development of a method to use standard hospital gamma cameras as triage whole body monitors in UK emergencies.

This paper outlines the process by which a medical gamma camera can be utilised to support assessment of internal radionuclides for the public. While hospital based gamma cameras are able to detect photopeaks, they are often limited to an energy range of 40-540 keV. However, radionuclides with photopeak energies above 540 keV can still be detected as the partial collection of photon energy increases the count rate at lower energies.

Discovery and Preclinical Profile of ALG-055009, a Potent and Selective Thyroid Hormone Receptor Beta (THR-β) Agonist for the Treatment of MASH.

Agonists of thyroid hormone receptor β (THR-β) decreased LDL cholesterol (LDL-C) and triglyceride (TG) levels in human clinical trials for patients with dyslipidemia. The authors present the highly potent and selective compound ALG-055009 () as a potential best in class THR-β agonist. The high metabolic stability and good permeability translated well in vivo to afford a long in vivo half-life pharmacokinetic profile with limited liability for DDI, and it overcomes certain drawbacks seen in recent clinical candidates.

Evidence-informed, experience-based co-design: a novel framework integrating research evidence and lived experience in priority-setting and co-design of health services.

Objective: To describe a new co-design framework termed Evidence-informed, Experience-based Co-design (E2CD).

Background: Involving consumers and clinicians in planning, designing and implementing services results in the end-product being more likely to meet the needs of end-users and increases the likelihood of their uptake and sustainability. Different forms and definitions of co-design have been described in the literature and have had varying levels of success in health service redesign.