March was Rare Disease Month at Pharm-Olam. We started off by reviewing an article that was published in Nature (read it here). From there, we examined the field of rare disease research (click here). We went on to look at the specific drug classes and development platforms that researchers use in rare disease research. We covered small molecules (access it here), antibody therapy (available here), protein replacement therapies (read it here), oligonucleotide therapies (read it here), gene therapies (click here to read it), and drug repurposing (available here).
Rare Disease Day may have been on 29 February, but at Pharm-Olam, we think it desires an entire month. Since the beginning of March, we have been focusing on rare disease research. We reviewed an article from Nature (click here to read it) and took a closer look at rare disease research as a whole (read it here). From there, we reviewed small molecules (click here), antibody therapy (access it here), protein replacement therapies (available here), oligonucleotide therapies (click here for the article), and gene therapies (read it here).
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Pharm-Olam is a mid-sized, global CRO that provides clinical research solutions for rare diseases, oncology, hematology, autoimmune conditions, allergies, and infectious diseases. Our organization was hired to support a Phase I, first-in-human study of a programmed cell death receptor (PD-1) inhibitor monoclonal antibody (mAb) drug treatment for solid tumor malignancies. The research is in its final stages. Pharm-Olam is proud to have supported this critical research, the patients and the sponsor involved.
Cohort management is part and parcel of conducting dose-escalation studies, but not all teams effectively manage it. Pharm-Olam is focused on expediting our sponsors’ early phase cycle time in Oncology to enable rapid delivery of a mean tolerated dose (MTD)/recommended Phase 2 dose (RP2D). Thus, tightly orchestrated cohort management strategies that both protect the safety of trial participants and support the capture of the quality data that sponsors need to move to the next stage of development are equally important.
Earlier this week, we looked at rare disease research in the broadest terms (read the article here). We explained that rare disease is a classification that is comprised of thousands of different conditions, but, collectively, the number of people with rare diseases is quite significant. Most of these conditions are caused by inheriting the right combination of genes to bring a recessive condition to the surface, but there are other possibilities, including illness and allergies. Understanding this root cause enables researchers to develop treatments.
Clinical research has grown in leaps and bounds. Researchers now have the tools to understand many of the world’s rarest diseases at a molecular level. At the same time, current legislation supports the development of treatments to address these rare conditions. However, challenges in this realm persist. A recent article in Nature reviewed the challenges in rare disease development as well as the opportunities that different rare disease development platforms present.
In this post, we discuss elements and lessons learned from a recent BARDA funded Influenza A Phase II study. The study’s purpose was to assess the efficacy of the Sponsors IP in treating severely ill and hospitalized Influenza A patients. Subject Detail: The study was targeted to enroll a subset of the general Influenza A global population. The study inclusion criteria required that each patient be ill for a certain number of days which could not be exceeded. The subject must also be hospitalized and specifically on oxygen therapy.