The new treatment for the central nervous system (lat. Systematis nervosi centralis) was developed by Lacerta Therapeutics. The company is a clinical-stage gene therapy company using a constellation of proprietary adeno-associated virus (AAV) vector technologies to develop treatments for the central nervous system and lysosomal storage diseases. With unmatched scientific expertise and depth in the development, production, and application of AAV vectors, Lacerta Therapeutics is advancing its clinical programs using its proprietary capsid variants and scalable vector manufacturing platform. The experience and accomplishments of the founders are unsurpassed in AAV gene therapy. Their AAV platforms allow early access to novel capsid variant identification and robust vector manufacturing.
Lacerta Therapeutics is combining rational and combinatorial methodologies in a state of the art AAV technology platform capable of directed evolution of novel capsid variants possessing selective vector characteristics. Depending on the selection pressure applied, the Lacerta Therapeutics approach allows for the development of novel AAV vectors with improved transduction, tissue- or cell subtype-selectivity, and immune escape profiles from a single highly complex library derived from any of the naturally occurring serotypes.
The Lacerta Therapeutics AAV library strategy has the following advantages over other combinatorial approaches. Firstly, their strategy results in capsid libraries that are logs better in complexity than less sophisticated, shuffled serotype libraries. Secondly, their strategy involves pre-selection for 3D compatibility using insight gained from thorough analysis of AAV structural characteristics and target only capsid variable regions (VRs) to combine mutagenesis and evolution in a unified approach.
A major hurdle to the broad application of AAV vectors to treat large clinical indications is the lack of a robust, scalable production platform. Industry-wide, much effort is being placed on the development of vector manufacturing platforms that ultimately will be insufficient to provide yields capable of supporting large clinical trials or in disease indications where commercial demand may make many of the current manufacturing systems obsolete. Moreover, many of the current systems require multiple, open manipulations, thus raising the risk of potential contamination of cultures and loss of clinical vector material.
Lacerta Therapeutics' OneBac insect cell-based production platform has many advantages over other vector production systems including Limited production components; Sf9 stable cell lines capable of growth in suspension to high volumes and cell densities. Robust AAV yields following infection with single, stable baculovirus expressing the disease-specific transgenes; Taken together, the Lacerta Therapeutics OneBac vector production platform significantly reduces the overall cost of goods while generating high yield, quality AAV vector preparations. The company has raised $30M of private investment. Still, it is actively seeking collaborative partnership and out-licensing opportunities providing potential partners with numerous advantages over current AAV vector platforms.