Sarepta & Codiak to research, develop exosome-based therapeutics

Precision medicine specialist, Sarepta Therapeutics, and Codiak BioSciences, a company at the forefront of advancing engineered exosomes as a new class of biologic medicines, are collaborating to design and develop engineered exosome therapeutics to deliver gene therapy, gene editing and RNA technologies for neuromuscular diseases.

The engineered exosome approach offers the potential to effectively deliver genetic therapeutics without triggering the adaptive immune response.

The two-year agreement includes up to five neuromuscular targets. Codiak is eligible to receive up to $72.5 million in upfront and near-term license payments plus research funding. Sarepta is granted an option to any of the candidates developed pursuant to the research alliance.

Exosomes are natural nanoparticles that serve as the body’s intercellular communication system, facilitating the transfer of a wide variety molecular payloads between cells.

As they are derived from human cells, exosomes provide a unique advantage as a targeted delivery system for genetic medicines because they are inherently non-immunogenic.

Through its proprietary, engEx Platform, Codiak can engineer exosomes with specific cargos and guide tropism to cell types of interest.

The collaboration will leverage Codiak’s exosome engineering capabilities with Sarepta’s expertise in precision genetic medicine to develop next generation therapeutics for patients with neuromuscular diseases that have few or no treatment options.

Under the terms of the agreement, Sarepta has the exclusive option to license Codiak’s technology to develop and commercialize engineered exosome therapeutics for up to five neuromuscular targets.

Sarepta and Codiak will collaborate to design exosomes that can deliver and functionally release select payloads, such as nucleic acids and gene therapy and gene editing constructs, in neuromuscular indications.

If Sarepta elects to exercise its option on a target, Codiak will be responsible for research and preclinical development through IND preparation, and Sarepta will be responsible for clinical development and commercial activities.

In addition to upfront, research funding and license payments, Codiak will be eligible to receive significant development and regulatory milestone payments and tiered royalties on future sales.

Gilead acquires equity interest in pioneering immunotherapy company

Gilead Sciences is acquiring a 49.9% equity interest in Pionyr Immunotherapeutics, a privately held San Fran company developing first-in-class cancer immunotherapies.

The acquisition – for $275 million – also provides Gilead with an exclusive option to purchase the remainder of the company.

Under the agreement, Pionyr’s shareholders may receive up to an additional $1.47 billion in option exercise fees and future milestone payments.

Pionyr’s Myeloid Tuning therapies have the potential to treat patients who currently do not benefit from checkpoint inhibitor therapies. PY314 and PY159 have demonstrated preclinical efficacy, suggesting potential in solid tumours in combination with established anti-PD(L)-1 agents.

Pionyr plans to file investigational new drug (IND) applications with the FDA for both PY314 and PY159 in the third quarter of this year. Pending Phase 1b results from either candidate – or sooner if Gilead chooses – Gilead can exercise its exclusive option to acquire the remainder of Pionyr.

“Pionyr is pursuing promising, novel biology in the field of immuno-oncology,” said Daniel O’Day, Chairman and Chief Executive Officer, Gilead Sciences.

“The agreement represents important progress as we continue to build out Gilead’s presence in immuno-oncology with innovative and complementary approaches.”

New class of precision medicine strips cancer of its DNA defences


A new precision medicine targeting cancer’s ability to repair its DNA has shown promising results in the first clinical trial of the drug class.

The new study, designed to test the drug’s safety, found that half of patients given the new drug either alone or with platinum chemotherapy saw their cancer stop growing, and two patients saw their tumours shrink or disappear completely.

Damage to the DNA in cells is the root cause of cancer – but it is also a fundamental weakness in tumours, and cancer cells can be killed by further damaging their DNA or attacking their ability to repair it.

The new phase I trial tested the first in a new family of drugs blocking a key DNA repair protein called ATR. Phase I trials are designed to assess the safety of new treatments, and it’s unusual to see a clinical response at this stage.

A team at The Institute of Cancer Research, London, and The Royal Marsden NHS Foundation Trust, led a trial of the benefit of an ATR inhibitor called berzosertib either on its own or with chemotherapy in 40 patients with very advanced tumours, treated in hospitals around the world.

The researchers established the doses at which the drug was safe for use in further clinical trials, and found berzosertib on its own caused only mild side effects.

Surprisingly for a phase I trial, the researchers also found that berzosertib stopped tumours growing in over half of patients given the drug either on its own or with chemotherapy – 20 out of 38 patients whose treatment response could be measured.

The drug’s benefit in blocking DNA repair was even more marked in patients also given chemotherapy, which works by causing DNA damage. In these patients, 15 of 21, or 71 per cent saw their disease stabilise – suggesting that chemotherapy boosted sensitivity to berzosertib.

The drug is now moving forward in further trials, and the hope is that it could be developed into a new targeted treatment for patients, and help overcome resistance to other precision medicines such as PARP inhibitors that target DNA repair.

The new results are published in the Journal of Clinical Oncology and the trial was funded by Merck, the manufacturer of the drug.