Tagged with Gene therapy

China While cell and gene therapies are all the rage now, a little known fact is that the world’s first approved gene therapy actually came from a Chinese company – in 2003! Gendicine® was a gene therapy for the treatment of haemophilia B. While China mostly lost the advantage of that…

Opinion Novartis Oncology’s Emanuele Ostuni (lead author) and Karin Blumer draw on their experience commercialising CAR-T therapies to make the case for the myriad benefits of collaborative, as opposed to command, leadership, the impact of company and team culture on the innovation journey, and why we must not confuse collaboration with…

China Following a recent capital injection of USD 67 million, Chinese biotech EdiGene is set to advance its pipeline in gene editing treatments and therapies as it becomes a clinical-phase company. The company’s CEO spoke to PharmaBoardroom about how Edigene’s technology platforms differ from those of other firms, and why the…

Japan Toshio Fujimoto, MBA, MD of Japan’s Shonan Health Innovation Park looks at how a technology developed in Japan over a decade ago is poised to drive major medical advances and new treatments.   Here’s what we know today and what we’ll need to do tomorrow to benefit from the promises…

USA Certara’s Ulrich Neumann looks at the challenges that payment models for cell and gene therapy (CGT) have faced, the multiple models now under development, and why an open and collaborative approach among US healthcare stakeholders is needed if the significant legal and practical barriers are to be overcome.   The…

Norway Photo: Sofia Linden / Oslo Cancer Cluster Oncologist Jon Amund Kyte shares some of the groundbreaking immunotherapy research that he and his team is conducting at Oslo University Hospital. Kyte also explores how Norway can better develop its clinical footprint and what his hopes for the future of experimental cancer…

Japan Dr Ken-ichiro Hata, representative director and chairperson of Japan’s Forum for Innovative Regenerative Medicine (FIRM) outlines the organisation’s mandate, how Japan has been able to cultivate one of the world’s most mature regenerative medicine ecosystems, and highlights key challenges including regulatory misalignment across Asia and the need for better manufacturing…

Novartis Ameet Mallik, Executive VP and Head of Oncology for the US at Novartis Oncology highlights the company’s culture of ‘curious, inspired, and unbossed,’ how Novartis differentiates itself in a highly competitive oncology landscape, learnings from recent product launches, and how Novartis is working to make its CAR-T therapy more accessible…

Cell & Gene Therapy The completion of the Human Genome Project (HGP) in April 2003 with the publication of the first human genome sequence is probably one of the most significant landmarks in humanity’s quest for knowledge, costing USD three billion across 13 years. But even after nearly two decades, we have barely scratched…

Novartis In late 2019 Novartis hit the headlines in Belgium after it refused to grant access to its one-off gene therapy Zolgensma® for the ultra-rare muscle-wasting disease spinal muscular atrophy (SMA) to Pia Boehnke, a Belgian child.   With Zolgensma® at that point having not yet been authorised by the European…

Opinion Pharmaceutical innovation is racing ahead of the US health system’s ability to adjust payment models, but innovative concepts are finally emerging. We will have to move beyond siloed thinking to establish them for the sake of getting patients sustainable access to a new wave of transformative cures, argues Ulrich Neumann.…

Rankings Below we present a list of the top 5 public biopharma companies that focus on developing RNA-based therapies. RNA interference (RNAi) is a natural mechanism which is present in living cells that selectively “turns off” specific gene’s expressions, thereby affecting the production of a specific protein that contributes to disease.…