Novo Nordisk and NanoVation Therapeutics have entered into a multi-year partnership worth $600m to advance genetic medicines for cardiometabolic and rare diseases.

The deal combines Novo’s capabilities in the two areas with NanoVation’s proprietary long-circulating lipid nanoparticle (lcLNP) technology for RNA delivery to cells outside of the liver.

The companies will work together on two lead programmes to develop base-editing therapies for certain rare genetic diseases, with the deal also covering up to five additional future targets for cardiometabolic and rare diseases.

Under the terms of the agreement, Novo will be granted a defined exclusive, global licence to use the technology for the two lead programmes, while NanoVation will receive research funding from Novo and will be eligible for up to approximately $600m in upfront cash and potential milestone payments, as well as tiered royalties on future product sales.

NanoVation’s lcLNP technology has already been shown in preclinical studies to deliver nucleic acids to multiple cell types beyond the liver, and demonstrated improved potency, safety and stability compared to conventional systems.

Eli Lilly and HAYA Therapeutics have entered into a multi-year agreement worth up $1bn to discover regulatory genome targets for obesity and related metabolic conditions.

The collaboration centres around HAYA’s proprietary regulatory genome discovery platform, which the partners will use to identify, characterise and validate multiple long non-coding RNA (lncRNA) targets for the potential development of new treatments.

In exchange, HAYA will receive an undisclosed upfront payment, including an equity investment, and will be eligible for up to an aggregate $1bn in preclinical, clinical and commercial milestone payments, as well as royalties on potential product sales.

Approximately 98% of the human genome is composed of non-protein coding regions. According to HAYA, this portion of the genome is dynamically active, generating thousands of therapeutically unexplored lncRNAs.

The company’s discovery platform enables the identification of tissue-, disease- and cell-specific lncRNA targets and the development of RNA-targeting therapies with “potentially better efficacy and less toxicity than current treatments”.

The Swiss biotech is already developing a pipeline of lncRNA-targeting precision therapies for the cell-specific treatment of a range of diseases, including heart failure.

Roche’s injectable monoclonal antibody Ocrevus Zunovo (ocrelizumab and hyaluronidase-ocsq) has been approved by the US Food and Drug Administration (FDA) to treat both relapsing and primary progressive multiple sclerosis (MS).

The subcutaneous (SC) formulation, which combines Ocrevus (ocrelizumab) with Halozyme Therapeutics’ Enhanze drug delivery technology, can be injected over approximately ten minutes while maintaining the same twice-yearly dosing schedule as the previously approved intravenous (IV) infusion.

Affecting more than 2.9 million people globally, MS is a neurological disease in which the immune system attacks the protective myelin sheath that covers the nerves and disrupts communication between the brain and the rest of the body.

The FDA’s latest decision on the therapy was supported by positive results from the late-stage OCARINA II trial, which demonstrated no clinically significant difference in the levels of Ocrevus in the blood when administered subcutaneously, as well as a safety and efficacy profile comparable to the IV formulation in patients with relapsing and primary MS.

Additionally, over 92% of patients who were surveyed as part of the study reported being satisfied or very satisfied with the SC administration.

Novartis’ Fabhalta (iptacopan) has been recommended by the National Institute for Health and Care Excellence (NICE) to treat paroxysmal nocturnal haemoglobinuria (PNH) in adults with haemolytic anaemia.

The factor B inhibitor will be available immediately on the NHS in England and Wales to treat patients with the rare blood disorder that affects approximately ten to 20 people per million worldwide.

PNH patients have an acquired mutation that causes them to produce red blood cells susceptible to premature destruction by the complement system, potentially leading to anaemia, thrombosis, fatigue and other symptoms that can impact quality of life.

NICE’s final guidance follows the Medicines and Healthcare products Regulatory Agency’s approval of the oral monotherapy in August and was supported by positive results from the late-stage APPLY-PNH trial in adults with PNH and residual anaemia despite prior anti-C5 treatment, which showed that patients who switched to Fabhalta experienced superior increases in haemoglobin levels compared to those who continued on anti-C5 treatment.

Results from the phase 3 APPOINT-PNH study in complement inhibitor-naïve patients also supported the decision.