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July 30, 2021/MedClub News/--Gene therapy can be said to be extremely hot at present, and many new possibilities have been developed for the treatment of many incurable diseases
.
However, existing gene therapies still face many challenges, from tissue specificity to low tolerability, to lack of efficacy and inability to re-administer drugs, which limit the implementation of gene therapy into the patient market
Ring Therapeutics completed a US$117 million Series B financing this time, hoping to face these challenges by introducing a new gene therapy vector platform
.
Series B financing came from Flagship, Invus, Altitude Life Science Ventures, Partners Investment, UPMC Enterprises, T.
Ring used the symbiotic virus group to create Anellovectors, an engineered vector composed of single-stranded DNA loops.
Once used, it will remain in the nucleus as an episome
In addition, once these capsidized viruses enter the cell, they still exist in the form of free single-stranded DNA molecules, and do not integrate with existing human DNA, and the risk is low
.
At the same time, this type of viral vector can live in harmony with the immune system in many different parts of the human body, and will be suitable for the treatment of many diseases
Tuyen Ong, MD, CEO of Ring Therapeutics said: "Because these ring viruses are immune to a certain degree, we have been able to prove in preclinical and other data that these vectors can be repeatedly administered
.
Ring virus vectors Gene therapy may solve the core problem in gene therapy, that is, having a viral vector that can be administered repeatedly
Dr.
Tuyen Ong said: "For thousands of years, the ring virus has been a part of the human body and constitutes most of the human symbiotic virus group
Since Ring was founded in 2017, it has been studying which viruses do not cause disease, so as to determine which viruses exist in and between humans
.
The company has discovered thousands of candidate vectors based on ring virus, and has the potential to achieve gene therapy and nucleic acid drugs in various diseases
Ring has not yet disclosed its first indication, which may be in the fields of central nervous system and cardiovascular diseases, as well as immuno-oncology and oncology
.
However, given that the viral vector does not have the common defects in other gene therapies, the field of disease treatment can be said to be very broad
.
Limitations of current gene therapy viral vectors
Currently, the vectors used in gene therapy are mainly viral vectors, including adeno-associated virus (AAV) and lentivirus, among which AAV vectors are the most commonly used and most stable introduction platform
.
Although AAV vectors are relatively mature and a variety of specialized types have been developed, there are also many challenges, such as:
First of all, as a virus, the safety of its production requires strict supervision;
Secondly, AAV virus has immunogenicity problems, may integrate with the host's genome, and has the risk of insertion;
Furthermore, the upper limit of the packaging of the viral vector is low, which is not friendly to some larger therapeutic gene fragments;
At the same time, the durability of its treatment is also a future challenge for this therapy;
The AAV viral vector can cause the immune system response in the patient, so it can only be administered once in a lifetime.
When the gene fragments with therapeutic effects are insufficiently replicated, repeated administration will not be able to increase the efficacy
.
In addition, this kind of drug may also cause potential liver toxicity, and so on
.
Non-viral vectors are also one of the new directions developed to solve the limitations of AAV vectors, such as plasmids, exosomes, red blood cells, and so on
.
Although non-viral vectors are superior to viral vectors in terms of safety and cost, there is currently no reliable data to prove their actual efficacy
.
I hope that in the future, there will be more new developments in the gene therapy vector platform
.
Reference source:
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