Our technology transfer team has helped the Medical Research Council (MRC), a founder in PepGen, to advance a novel technology that could improve the effectiveness of cutting-edge nucleic acid therapeutics.
When Dr Mike Gait’s lab of the MRC Laboratory of Molecular Biology in Cambridge in Cambridge and Professor Matthew Wood’s research team at the University of Oxford created a series of novel and innovative cell-penetrating peptides to enhance the delivery of therapeutic cargos to previously inaccessible tissues and intracellular targets, LifeArc’s Technology Transfer Team was on the journey with the scientists every step of the way.
LifeArc’s work has supported the scientists to progress the technology from the lab bench to the point now where it is ready to move towards the clinic.
Working in partnership
To realise the potential of this research, PepGen was set up in 2018, a spin-out arising from the MRC and the University of Oxford’s research.
LifeArc had been working closely with the University of Oxford and Oxford University Innovation over several years to secure intellectual property (IP) rights and build a robust patent portfolio focused on the cell-penetrating peptides. Over a period of two years, LifeArc negotiated on behalf of MRC the contracts concerning the licensing, setup, and Series A funding round of PepGen.
PepGen is focused on advancing next-generation nucleic acid therapeutics for Duchenne muscular dystrophy (DMD) and other severe neuromuscular conditions into the clinic.
The startup has now closed a total $45m investment led by RA Capital with participation from Oxford Sciences Innovation (OSI), the University of Oxford and CureDuchenne Ventures. This funding will help take lead candidate programmes through early-phase clinical trials.
Dr Caroline Godfrey, CEO of PepGen said: “PepGen’s technology was developed over the course of a 10-year collaboration between Dr Michael Gait and Prof Matthew Wood from the University of Oxford. We are enormously grateful to UKRI and LifeArc for their support during the spin-out process, and across the development of the company to date.”
LifeArc continues to play a key faciliatory role, representing MRC as a board observer and closely monitoring the development of MRC IP up to the next funding round.
Delivering treatments into cells
Nucleic acid therapeutics are designed to treat the underlying cause of a genetic condition. However, one major stumbling block to the success of this approach in the clinic is the failure of nucleic acids to get to key tissues and reach their site of action within the cell.
One of PepGen’s lead candidates is a peptide-conjugated nucleic acid therapeutic for DMD, which is designed to help overcome a specific type of fault within the dystrophin gene. This fault leads to the body not producing enough – or any – of the protein dystrophin. It is the absence of this protein which leads to progressive muscle weakness. Most children born with this condition do not live beyond early adulthood as the muscle weakness leads to heart failure or severe respiratory complications.
The core platform technology licensed to PepGen is based on cell-penetrating peptides conjugated with a splice-switching antisense oligonucleotide to restore the expression of the absent protein. Once inside the cell, the technology encourages the cell machinery to ‘skip over’ the affected part of the gene – allowing the body to make a shorter form of the dystrophin protein that is still functional.
Looking to the future
PepGen’s unique competitive position is that the peptide vector enables delivery to heart, among other key tissues – a challenge that few therapies can address.
New PepGen headquarters will be created in Boston (US), from where late-stage pre-clinical testing, clinical and regulatory functions will be managed. The UK base in Oxford remains the hub for early-stage research.
Find out more: pepgen.comAll Case Studies