Gene therapy - Lipid nanoparticles

"Gene Therapy: Lipid nanoparticles" is part of the analyst's Analyst opinions series. Gene therapy sales are projected to exced $10bn by 2027 and lipid nanoparticles (LNPs) have gained popularity as carriers for gene therapies due to their high tolerance, low toxicity and limited side-effects, which make them prime candidates for treatment of a vast range of diseases such as genetic and autoimmune, disorders, cancer, and infectious diseases. LNPs hold advantage over other types of vectors - e.g. viral, bacterial, plasmid etc. - since they induce minimal immune responses and exhibit enhanced physical stability, which make them suitable for repeated and sustained administration of therapies.

This report discusses the developing trend and substantiates the assessment by analysing signals and trends from various alternative datasets, including funding activity, clinical trials, and drug databases, all of which underscore the transformative potential of LNP-based gene delivery technology. The report further highlights the competitive landscape and identifies leading innovative companies and startups from M&A perspective.

Key takeaways from the report:

Advancement of LNPs

Lipid nanoparticles (LNPs) have evolved from earlier generations of targeted drug delivery systems, demonstrating high efficiency in delivering antibodies, targeted drugs, and prodrugs. The mRNA-LNP combination is particularly well adopted for immune system.

Role in Gene Therapies and Vaccines

The adoption of LNPs as a preferred non-viral vector has surged, particularly in gene therapies and mRNA vaccines against SARS-CoV viruses, due to their proven ability to deliver stable mRNA compositions to targeted sites. LNPs were crucial in the successful deployment of mRNA vaccines during the COVID-19 pandemic.

Emerging innovation

Patent analysis by Technology Foresights indicates that LNPs in gene therapies represent a high-impact innovation. The increasing rate of patent filings highlights LNPs as an emerging technology within the pharmaceutical industry. Patenting activity for LNPs is growing at a 60% CAGR over the past three years, with the US and China leading as innovation hubs. Large pharmaceutical companies dominate the patent landscape, while startups contribute approximately 10% of all patents, focusing on oncology, neurological disorders, and cardiovascular diseases.

Investment Surge

Investment in LNP delivery platform startups is increasing, with major companies like BMS, Eli Lilly, and Amgen investing as recently as 2024. Pfizer, Merck, and Takeda Pharma have also formed strategic partnerships for co-developing LNP delivery technologies with key IP holders.

Drugs and clinical trials

Moderna and BioNTech among leading sponsors of clinical trials. Metabolic disorders and cancers among leading indications for drugs. As many as 15 therapies in advanced clinical trials with robust pipeline of new therapies building up YoY.

Innovation landscape and M&A opportunities

Moderna, Sanofi, and BioNTech are among the top innovators out of 60+ large companies. Other significant players in gene therapies are developing in-house LNP platforms or collaborating with other entities. Over 50 startups, including Oisin Bio, ORNA Therapeutics, and eTheRNA, are at the forefront of LNP innovation. The startup leadership map provides valuable insights into the robustness of innovation portfolios and opportunities for potential partnerships, mergers, acquisitions, and other investments.

Scope

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