New therapeutic modalities are proliferating
During the last thirty years, the number of therapeutic modalities with entirely new mechanisms of action has proliferated. Not only have they expanded the number of treatable diseases, but they have also improved efficacy and safety. In 2023, more than 25% of clinical trials were harnessing new therapeutic modalities.
New Therapeutic Modalities are Proliferating3
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Precision therapies could reverse the downtrend in returns on R&D
Given regulatory bottlenecks and legacy drug discovery methods, the return on therapeutic Research and Development (R&D) has been falling for nearly 25 years. This has increased the hurdle rate for new drug discovery R&D. But, according to the research of our accredited genomics team, novel therapeutic modalities and R&D methods, coupled with regulatory approval of precision therapies, could reverse this downward trend in return on investment in the pharmaceutical industry.
Average Annual R&D and Incremental Revenue Attributable to Drugs Released4
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The value of curing rare diseases like sickle cell anaemia is high
These advances in could accelerate the pace of scientific drug discovery, personalising medicine to cure disease instead of simply masking its symptoms. Among precision therapies, gene editing treatments like CRISPR-Cas9,have the potential to cure rare genetic diseases such as Sickle Cell Disease (SCD) and Beta Thalassemia. SCD is an inherited red blood cell disorder that affects more than 100,000 people in the US and 20 million people globally, primarily in Africa. Today, therapeutics account for ~16% of the total spent on treating SCD disease in the United States, but they have done little more than manage symptoms, as the life expectancy of SCD patients is only 56% that of the general population.
SCD Healthcare Costs Over Average Patient Lifetime and Reasonable Cost for Sickle Cell Disease Cure5
![The value of curing rare diseases like sickle cell anemia is high](https://europe.ark-funds.com/wp-content/uploads/2024/04/The-value-of-curing-rare-diseases-like-sickle-cell-anemia-is-high-1024x389.jpg)
In 2023, however, the medical community achieved a milestone with the first approvals for gene editing treatments for SCD and Beta Thalassemia in both the United States and Europe, signalling a new dawn for patients burdened by these conditions.6 Meanwhile, in the UK in 2022, a young leukaemia patient found hope through a pioneering T-cell therapy, marking another step forward in our battle against cancer.7
Wright’s Law* predicted the cost declines in proteomics
Mass spectrometry is an analytical technique that identifies the composition of a substance by ionising proteins and measuring their mass-to-charge ratios. As the number of proteomes analysed using this approach has increased, the costs associated with mass spectrometry have dropped significantly. This has unlocked new possibilities in medical research and diagnostics. Our research suggests that for untargeted Proteomics using mass spectrometry, the cost per sample is declining 23% at an annual rate, or ~11% for each cumulative doubling in the number of proteomes sequenced. This aligns with Wright’s Law which predicts that for every cumulative doubling of units produced, costs will decrease by a fixed percentage.
Proteomic discoveries are also paving the way for the identification of novel biomarkers, which are measurable substances in an organism whose presence indicates some biological state or condition. This in turn enables the earlier detection and treatment for unique cancer subtypes.
Wright’s Law has Predicted the Cost Decline for Untargeted Proteomics and US Trails with Patient Biomakers8
![Wright's law has predicted the cost decline of proteomics](https://europe.ark-funds.com/wp-content/uploads/2024/04/Wrights-law-has-predicted-the-cost-decline-of-proteomics-1024x398.jpg)
Single-cell RNA sequencing is revolutionising our understanding of cancer
RNA sequencing is a high-throughput sequencing technique used to capture and sequence the complete set of RNA transcripts in a sample, providing insights into gene expression patterns and functional analysis of genes at a given moment. This approach is particularly helpful in developing our understanding in gene expression between healthy and cancerous cells, identifying potential biomarkers for diagnosis and uncovering targets for new treatments. While traditional gene expression analysis using RNA sequencing can measure only the expression of genes in a mixture of different cell types, single-cell RNA sequencing can delineate the expression of different cell types in a complex tissue sample. Theoretically, linking gene expression to specific cells increases the accuracy of measuring by 10x and cuts costs per gigabyte by 76%. In other words, single-cell RNA sequencing provides a more detailed view than traditional RNA sequencing, revealing the cellular heterogeneity within tissues, including tumors that could be masked in bulk RNA sequencing analyses.
Process of the RNA Sequencing9
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