New trends in the biotech industry Flashcards
Biotech Evolution, Kendall Square, Gene Therapy, Orphan Drug Act, Therapeutic Delivery Challenges, Entrepreneurship in Biotech
What is the significance of Kendall Square in biotechnology?
Biotech hub known for innovation and research.
What characterises the current trend in biotech with regards to the different classes of therapeutics?
A shift in focus from small molecules and antibodies to cell and gene therapy.
What is gene therapy?
A technique for correcting defective genes responsible for disease development.
What are the approaches used in gene therapy?
Gene replacement, silencing, addition, and editing.
What are the challenges for gene therapy?
Restoration of a defective gene may not be sufficient to cure some diseases, too few patients for clinical trials for rare diseases, challenges with delivery of nucleic acids.
What challenges are associated with therapeutic delivery of nucleic acids?
For viral vectors: immune responses, limited payload capacity, risk of insertional mutagenesis, manufacturing complexities.
For lipid nanoparticles: limited biodistribution, toxicity, low efficiency of endosomal escape.
What are the advantages of red blood cell-derived extracellular vesicles as gene delivery vehicles?
Efficient delivery, safety/biocompatibility, scalable and sustainable manufacturing, favourable biodistribution.
What is the Orphan Drug Act?
Legislation to encourage development of drugs for rare diseases by providing tax credits, market exclusivity, application, reduced statistic requirement, etc.
What are key factors for entrepreneurship in biotech?
Business aspect: business plan and investment
Idea aspect: innovation and IP
Team aspect: leadership, , R&D team, and scientific advisory board.
Infrastructure aspect: lab space and conducive regulations
How does gene editing differ from traditional gene therapy?
Gene editing involves permanent alterations to the genome.
How do immune responses impact gene therapy?
Can limit effectiveness and safety of treatments and prevent redosing.
What are the delivery methods for gene therapy?
Viral vectors, lipid nanoparticles, extracellular vesicles.
Which types of viral vectors are commonly used for gene therapy delivery?
Lentiviruses (LVs) and adeno-associated viruses (AAVs).
Why are lipid nanoparticles important in biotech?
They enable the delivery of mRNA vaccines and gene editing tools.
What challenge does the biotech industry face with delivery hurdles?
Ensuring targeted and efficient delivery of therapies to specific tissues.
