Boryung Corp.

Drs. Dimmeler, Giacca, and Rayner spoke on the last day of the European Society of Cardiology (ESC) Congress 2024 about the promise of RNA therapies for cardiovascular diseases. Image of participants at a previous ESC Congress. Image: Shutterstock/ hydebrink On the last day of the European Society of Cardiology (ESC) 2024 Congress (2 September), a panel discussed the therapeutic potential with non-coding RNAs in cardiovascular disorders. Dr. Stefanie Dimmeler from Goethe University Hospital opened with a talk on her team’s work investigating the microRNA miR-92a and its inhibition. Dimmeler described her work in which antimiR-92a was observed to bind and block miR-92a. AntimiR-92a “improved blood supply and function in myocardial models,” and Dimmeler added that the potential therapeutic was anti-atherosclerotic, promoted endothelial cell growth, increased  wound healing, and reduced metabolic syndromes. A completed Phase I dose escalation study (NCT03494712) showed that miR-92a can be blocked by antimiRs in humans with a single injection IV with the compound, and this is associated with a change in the derepression of the [miRNA] targets, said Dimmeler. Next, Dr. Mauro Giacca from Kings College London, discussed strategies for delivering therapeutic RNAs in the cardiovascular setting. He stated, “I am among those who are strongly convinced that these molecules can somehow fill the pharma innovation gap.” He was referring to the gap between growing investment in the pharmacological industry and shrinking numbers of new drugs. Giacca explained his laboratory performed high throughput screenings to find RNAs with therapeutic potential in cardiovascular disease. He noted his team uncovered a series of siRNAs and miRNAs that arepowerful at driving proliferation. See Also: BR-6002 by Boryung Pharmaceutical for Duodenal Ulcer: Likelihood of Approval BR-6002 by Boryung Pharmaceutical for Gastric Ulcers: Likelihood of Approval “There are many applications that are amenable to these RNAs,”Giacca said, including cardioprotection, cardiac regeneration, cardiac gene editing, and cardiac cell trans-differentiation. He also described his research into delivery systems for such therapies, centred on his team’s success with lipid nanoparticles. Finally, Dr. Katey Rayner from the University of Ottawa discussed her work dissecting the role of miR-223 in regulating atherosclerosis. “The presence of miR-223 seemed to promote cholesterol efflux, and the absence of miR-223 seemed to inhibit cholesterol efflux,” Rayner stated. The miRNA also influences inflammation. Rayner said her team has shown that delivering miR-223 can be an effective way to remove cholesterol, and that atherosclerosis is much worse when in the absence of miR-223. With ribosomal fractionation, the team found, “there’s a number of proinflammatory genes that are being targeted by miR-223”, according to Rayner. In their panel discussion, the researchers emphasised the therapeutic potential of RNAs. But, as Rayner noted, “the risk of multiple targets – that’s a challenge that hasn’t been overcome.”

The Mpox treatment centre at Nyiragongo General Referral Hospital, north of Goma in the DRC. Credit: GUERCHOM NDEBO / Getty. As the number of mpox-related deaths in the Democratic Republic of Congo (DRC) and neighbouring countries continues to climb , international cooperation is needed to recognise the global risk posed by infectious diseases, and “to deal with them on a global basis.” That is according to Dr Marc-Alain Widdowson, the high-threat pathogens lead at the Europe branch of the World Health Organization (WHO), who was speaking on GlobalData’s Thematic Intelligence podcast . Widdowson noted the WHO’s calls for a pandemic treaty and pointed to both the “moral” and “self-interested” benefits to a global approach to the current outbreak of the clade 1b strain – a more severe variant affecting both adults and children. “There’s a dual responsibility that governments need to think about,” he explained. “I understand the electoral pressure to worry about what is going on within your own borders – and that is understandable to a point – but it is essentially, at the end of the day, a bit self-defeating, because if you don’t tackle the problem outside your walls then eventually they will get breached.” Widdowson spoke alongside Bronwyn Nichol, senior officer of public health in emergencies at the International Federation of Red Cross and Red Crescent Societies (IFRC). She pointed to the challenges associated with stopping the spread and highlighted the need for international cooperation to provide sufficient mpox vaccinations to affected communities. See Also: BR-6002 by Boryung Pharmaceutical for Duodenal Ulcer: Likelihood of Approval BR-6002 by Boryung Pharmaceutical for Gastric Ulcers: Likelihood of Approval “There have been pledges of vaccine stocks to be delivered to Africa, but it is less than 15% of what we need,” Nichol said. “One of the vaccines is a two-dose regimen, so that also means more is needed if we’re going to be able to deliver. Besides just the vaccines themselves, there’s also all the support that needs to go in, whether it’s cold chain management, the logistics of moving the vaccines, making sure that they’re accepted by communities.” An estimated 10 million mpox vaccines are needed across Africa, and some communities are particularly at risk. The WHO highlights the need for vaccines for healthcare workers; those living with mpox-infected relatives or partners; those with multiple sexual partners; and sex workers and their clients. A global mpox response will require international cooperation, but lessons should have been learnt from previous experiences, including from the Covid-19 pandemic. However, according to Widdowson, memories are short, and governments are too quick to become comfortable once immediate threats have passed. “The lesson that we keep on learning and forgetting is that infectious diseases are unpredictable. We’ve always got to remember that we can never be happy with the status quo. We’ve always got to think that things are going to blow up in our faces … what we haven’t really learnt from Covid is that, for us all to be safe, the disease needs to be tackled globally. The lesson from Covid really was that something that happens in one part of the world will very quickly affect the rest of the world.”

The trials enrol patients with type 2 diabetes and chronic kidney disease (CKD). CKD can be a complication of type 2 diabetes. Credit: NTshutterth via Shutterstock. ProKidney is dropping an ex-US Phase III study for its chronic kidney disease (CKD) cell therapy rilparencel, following discussions with regulatory experts and ex-US Food and Drug Administration (FDA) officials. The US-based biotech’s Phase III program consisted of two studies; the REGEN-006 (PROACT 1) (NCT05099770) study in the US and the REGEN-016 (PROACT 2) (NCT05286853) study in ex-US countries. Both trials are designed to investigate the agent, which is also known as REACT, in type 2 diabetes and CKD. Following discussions to finalise rilparencel’s regulatory plans in the US, North Carolina-based ProKidney will drop the REGEN-016 trial, a move which the company says will save between $150m and $175m. The company closed a $140m public offering of class A ordinary shares in June 2024, which would extend its cash runway to mid-2026. Rilparencel, which was granted regenerative medicine advanced therapy (RMAT) designation by the FDA in October 2021, is a regenerative therapy. Administered through intra-renal injection, it utilises autologous renal cells embedded in a gelatin-based hydrogel. These cells are designed to stimulate the regeneration of functional kidney tissue, aiming to restore kidney function based on organ regeneration technology. Enrolment into the REGEN-006 study was put on hold last year after ProKidney announced that it was updating the protocol. The study, which previously enrolled patients with mild-moderate to severe CKD, was updated to focus on patients with the most severe CKD. The two trials were expected to enrol up to 600 patients each. Subjects were randomised 1:1 into either a rilparencel treatment arm and undergo a kidney biopsy, or a sham control arm. See Also: BR-6002 by Boryung Pharmaceutical for Duodenal Ulcer: Likelihood of Approval BR-6002 by Boryung Pharmaceutical for Gastric Ulcers: Likelihood of Approval In the announcement accompanying the update, ProKidney’s CEO Bruce Culleton said: “We are confident that this strategic shift in our Phase III programme is the most expeditious and resource efficient approach to bring rilparencel to market in the US, our highest priority market.” Free Whitepaper Optimise your cell therapy process: a guide to cell thawing Typically carried out at the point of care, errors in cell therapy thawing could compromise treatment efficacy, leading to significant patient impact as well as high costs and a compromised reputation for the product’s developer. This guide addresses how cell thawing has historically developed into the new techniques used today, along with the physical and biological implications of key metrics and components such as warming rate and ice structure. Also included are reviews of key studies from scientific literature and a consideration of the interactions between cooling and warming rates, as applicable to cell and gene therapies. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic By downloading this Whitepaper, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy