Richa Madan Lomash, Ph.D., is a Therapeutic Development Project Manager at the National Center for Advancing Translational Sciences (NCATS), NIH, where she manages IND-enabling drug development programs for rare diseases across multiple modalities, including AAV gene therapies, peptides, small molecules, and antisense oligonucleotides. She serves as co-lead of the multi-institute Platform Vector Gene Therapy (PaVe-GT) program, advancing innovative gene therapy approaches toward clinical testing, and plays a key role in regulatory strategy, including FDA interactions, IND submissions, and orphan drug designations. Richa has contributed to NIH-wide initiatives such as RARe-SOURCE and i3D-RARE, aimed at accelerating translational science and therapeutic development. She is actively participating in gene therapy strategy groups, international rare disease consortia, and cross-sector collaborations with academia, industry, and patient advocacy organizations. A recipient of multiple NIH and NCATS Director’s Awards, she is also committed to mentorship, scientific dissemination, and advancing collaborative models to improve outcomes for patients with rare diseases. Richa trained as a cell biologist and did her postodoral training at NIH in the field of neurobiology undertsanding brain proteins.
This workshop session covers translational research initiatives that advance drug development for rare diseases. We will highlight pre-clinical testing strategies, and the tools and technologies that help move promising assets toward the clinic. We will also discuss pre-clinical platforms for AAV gene therapy, stem-cell-derived 3D models, and bioinformatics efforts to consolidate rare disease information. Participants will learn from concrete examples and get familiarized with resources available to advance their own rare disease research and development efforts.
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