The Success of Cell and Gene Therapy
There has been a strong resurgence in the fields of Cell and Gene Therapy over the past few years, driven in part by some major success stories in diverse indications, ranging from the successful treatment of blindness using new viral vectors to the emergence of Adoptive T-Cell therapy as an effective treatment for certain lymphomas. Off the back of these successes an enormous amount of capital has flooded back into the sector as companies seek to exploit recent advances.
The three challenges in this arena have always remained constant: delivery, expression and immunity. Novel vector systems need to be developed so that the target gene can be delivered to a sufficient number of cells in order that it can elicit a therapeutic effect. Likewise, new promoters need to be isolated that drive efficient, yet finely tuned, gene expression so that the therapeutic effect will last during the lifetime of the individual. Finally, given that the target gene is often delivered using immunogenic vectors (both viral and cellular) and that the therapeutic gene is often new to self, long-term immune responses to the therapeutic cassette need to be minimised.
Synpromics' Technology Enhances Gene Therapy
The technology created by the Synpromics team allows tighter control of gene expression and addresses each one of these issues, facilitating the creation of novel promoters tailored to:
- Tissue-type, e.g., liver, muscle, brain
- Developmental stage, e.g., lineage specificity in stem cells
- Cell affliction, e.g., pathogen-infected, diseased
- Inducible by environmental stimulus, e.g., temperature, chemical or biological
- Eliminate off-target gene expression
By enhancing the specific activity of the promoter that drives expression of the therapeutic gene it should prove possible to reduce vector dose, thereby increasing the safety profile of the therapeutic, lowering immunogenicity and reducing off-target side effects.
A Promoter for Any Cell Type or Condition
Synpromics generates panels of tailored promoters, each with unique sequences and each driving expression to a defined level. This confers increased stability onto the therapeutic product as multiple genes can be delivered by single vectors into a cell, controlled by unique promoters, thus preventing instability through recombination.
Promoters can be designed of any length, which is important for vectors based on the adeno-associated virus, which has a very short genome and consequently reduced capacity for the therapeutic cassette. Smaller promoters means that there is more space for the transgene and other important elements required for optimal control of its expression.
Finally, Synpromics creates promoters with unique sequences that do not exist in nature and are in essence man-made. This improves the patentability of any therapeutic construct comprising a Synpromics promoter, as the technology facilitates the discovery of promoters consisting of unique combinations of regulatory elements with the desired activity.