Less than two months after Peter Kolchinsky and Raj Shah announced a new $461 million fund, the partners at RA Capital Management appear to have made another investment.

RA is headlining a $45 million Series A round for the Oxford, UK-based biotech PepGen, which focuses on severe neuromuscular diseases like Duchenne muscular dystrophy. The company will use the funding to advance a slate of what theyre calling cell-penetrating peptides combined with some of their proprietary conjugates into the clinic.

We believe PepGens PPMOs have enormous potential for the treatment of severe neuromuscular and cardiac disorders, RA venture partner Ramin Farzaneh-Far told Endpoints News in an email. The financing reflects our confidence, and that of our syndicate partners, in the technology.

Oxford Sciences Innovation, PepGens seed investor, also participated in the round, as well as the University of Oxford and CureDuchenne Ventures. Wednesdays cash will also allow PepGen to build out a corporate team in the new Boston headquarters and expand the R&D hub in the UK, Farzaneh-Far said.

The move from RA comes shortly after Shah told Endpoints News in October that the cash for its Nexus I life sciences fund, roughly $300 million, was churned through at a relatively rapid pace. In just 15 months of investment, RA had spent about 80% of their fund, which prompted the Nexus II raise.

Though the new fund built off largely the first, the cash pools remain separate. Farzaneh-Far declined to comment to which Nexus fund Wednesdays investment belonged.

PepGen itself was spun out of Oxford in 2018 in order to further develop the peptides at the heart of its research. The biotech says that the cell-penetrating nature of the peptides, when conjugated with phosphorodiamidate morpholino oligomers or PPMOs, could allow for enhanced delivery of oligonucleotides to key tissues, while also improving safety compared to other medicines.

Specifically, PepGen is hoping to leapfrog the exon-skipping approaches already available in order to restore dystrophin expression in DMD patients, CEO and co-founder Caroline Godfrey said in a statement.

One of the areas where PepGen says its programs are beneficial is in the cardiovascular comorbidities that often accompany DMD. Because the peptides can penetrate cells, the company says its drug candidates strongly distribute to cardiac tissue.

With the recent approvals of treatments that generate small increases in dystrophin in skeletal muscle, patients may be ambulating and living longer, but this in turn is expected to shift the burden of morbidity and mortality towards an epidemic of heart disease, which is not adequately addressed by current DMD therapies, Farzaneh-Far said in an earlier statement.

This past summer, the FDA green-lit the third DMD drug when Japanese developer NS Pharma gained an accelerated approval for viltolarsen. That followed a wild back-and-forth between regulators and Sarepta, who originally rejected their DMD candidate in August 2019 but reversed course later that year.

The agency, however, still doesnt have full efficacy data on any of the three approved DMD drugs, as the OKs were all based on the same disease biomarker.

See original here:
RA Capital backs Oxford spinout PepGen with a $45M Series A, seeking to treat Duchenne and other similar diseases - Endpoints News