Recent advancement in the development of novel platforms of smart targeting small molecules, gene therapy, stem cell therapy, gene modification, and gene rectification technologies pose new prospects for therapies and cures for rare and genetic diseases. More than 80% of rare diseases are genetic in origin, so that gene and cell therapies are being proposed to treat them. Moreover, human stem cell therapy is suggested as one of the most promising alternatives.
Furthermore, genetically modified stem cells and autologous induced pluripotent stem cells, synergistically coalesced with zinc-finger proteins and transactivator-like effector nucleases, are targeted as the selection of choice for the ex vivo repair of specific mutated deoxyribonucleic acid (DNA) sequences.
Similarly, patient specific induced pluripotent stem cells can be edited and shifted back into the patient, leading to a new approach to precision medicine. In addition, once homed, stem cells secrete a vast number of paracrine factors that could become new therapeutic tools in the treatment of rare and genetic diseases.
Therefore, next-generation small molecules, gene therapy, stem cell therapy, and gene editing for DNA repair are promising approaches to the treatment of rare and genetic diseases. This research service (RS) depicts the current landscape and the new trends in the development of novel therapies for rare and genetic diseases, focused on highly interrelated and synergistic approaches.
Key Topics Covered:
1. Executive Summary
2. Technology Snapshot and Trends
3. Technology Status Review and Assessment
4. Impact of Rare Disease Therapies on the Pharmaceutical Industry
5. Intellectual Property Analysis
6. Funding and Investment Landscape
7. Technology Roadmapping
8. Performance Benchmarking and Strategic Recommendations
9. Key Industry Influencers
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