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Drug Discovery and Development

Rare Dermatologic Disorders Medication Development

Rare Dermatologic Disorders Medication Development refers to the specialized pharmaceutical research and innovation pipeline focused on diagnosing, treating, and managing uncommon skin diseases. It integrates precision medicine, biologics, and orphan drug strategies to address unmet clinical needs, accelerate regulatory pathways, and deliver targeted therapies for patients with complex cutaneous conditions.

Solutions
BioMendics: Where Science Meets Compassion
BioMendics
Where Science Meets Compassion
Karen McGuire, CEO and Co-Founder
In the world of rare diseases, few conditions are as physically and emotionally grueling as epidermolysis bullosa simplex (EBS). Often diagnosed in infancy, EBS causes fragile skin to blister and tear at the slightest touch, leading to chronic or recurring wounds, intense pain, and a lifetime of limitations. For the estimated 35,000 people in the U.S. living with EBS, there is still no FDA-approved, disease-modifying treatment.
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State of Industry

Unlocking Dermatologic Innovation Through Drug Repurposing

Dermatologic therapy is in a transformative shift from the protracted and expensive paradigm of de novo drug discovery toward more efficacious methodologies. The conventional model, despite its successes, often overlooks patients with rare or specialized cutaneous conditions, as the small market size cannot justify the substantial investment required. Presently, a more agile, intelligent, and efficient strategy is materializing: drug repurposing. This approach, which entails identifying novel therapeutic applications for existing, approved molecular entities, is rapidly emerging as the primary driver of innovation in underserved dermatologic indications. This strategy is not predicated on serendipity; rather, it represents a deliberate and scientifically rigorous undertaking. By leveraging established molecules, developers can circumvent the initial, most uncertain, and most capital-intensive phases of research, thereby creating a streamlined trajectory for introducing novel therapies to clinical practice. It unequivocally affirms the principle that the biological narrative of a single molecule extends beyond its initial approved indication.

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Deep Dive

Preserving Biological Fidelity in Tissue Dissociation

Tissue dissociation has become a much more important step in modern single-cell research than many laboratories once assumed. As scRNA-seq, flow cytometry and high-resolution cellular profiling become more central to translational and discovery research, scientists are paying closer attention to what happens to a sample before sequencing or analysis even begins. Increasingly, the concern is not just whether enough cells can be recovered, but whether the dissociation process itself changes the biology researchers are trying to measure.

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Leadership Perspective
Rapid Release Quality Control Solution for Cellular Therapies using Celsis ATP Amplified Bioluminescence
Charles River Laboratories
Rapid Release Quality Control Solution for Cellular Therapies using Celsis ATP Amplified Bioluminescence
Jon Kallay, Senior Technology and Market Development Manager

Quality Control Testing of Cellular Therapies

Cell and genetherapies have been gaining a lot of attention in the medical and research communities. They offer customized medicine to individual patients, but the life-saving capabilities of these products come with an increased level of complexity. They must be manufactured safely, and individual batches must be tested to ensure they were created to specification.

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