Bio Foundry
Manufactoring materials for Cell and Gene therapy made as per your needs.
Manufactoring materials for Cell and Gene therapy made as per your needs.
Successful clinical translation of viral vectors has created therapeutic opportunity for seemingly most life-threatening diseases. While therapies for monogenic disorders are majorily provided by the viral vectors, many other diseases such as cancer and metabolic disorders have shown successful clinical outcome with viral vector-based therapies. Most promising among the viral vectors are those based on lentiviruses and adenoviruses.
Lentiviral vectors are currently in different phases of clinical trials for the treatment of various genetic disorders when conjugated with ex vivo cell therapies. The lentiviral vectors have a long history of development to overcome the safety concerns. Currently, the third and fourth generation lentiviral vectors are based on the genetic components derived from HIV genome. With rational design and robust screening, third generation lentiviral vectors have overcome the safety concerns and other regulatory challenges. At present, most of the cell-based therapies use third generation lentiviral vectors. We at MicroCRISPR has developed an in-house vector manufacturing unit, which provides lentiviral vectors for gene therapies. Our lentiviral system uses both adherent and suspension cell culture systems with multi-step automated purification process, quality check, screening assays, dose optimization, purity analysis and efficacy assays.
Adeno-associated virus (AAV)-based therapies are have significantly grown over the years. The advantage of AAVs over other viral therapies is that they do not integrate into the genome and remain as episomal expression vectors in the cells. Their non- integrating property makes them unique for the delivery of genetic material into the replication deficient cells such as neurons. At present, a large number of clinical trials are ongoing utilizing AAVs for the treatment of neurological complications such as neurodegenerative diseases and other neurological complications. At MicroCRISPR we have engineered next generation AAVs for the treatment of neurological disorders such as Alzheimer’s disease, Parkinson’s disease, Amyotrophic lateral sclerosis and spinal muscular atrophy. We are also expanding our AAV therapeutic platform for the development of therapies for Hemophilia A, and Ocular diseases.