Nature Reviews Drug Discovery has an interesting article on "waves of drug discovery" that will soon occur in genomic medicines (despite past cycles of hype and disappointment.) The article is by Xiw, Chen, Wong, at Boston Consulting Group. If you look it up, don't miss the elaborate and data-backed appendix.
Here's the link (subscription):
On a less optimistic note, the same journal recently published an article on "herding" or copy-cat behavior, in drug development pipelines. Fougner et al.
Below, I clip some introductory material from Xie et al. on the genomic waves to come.
Genomic medicines: the coming waves?
"Genomic medicines have gone through periods of hype and disillusionment since their conceptualization. For example, the approval of the pioneering gene therapy alipogene tiparvovec (Glybera) a decade ago fuelled expectations of a subsequent wave of other virally delivered gene therapies. However, these expectations have not yet been realized, and with ~30 setbacks for genomic medicine candidates in the past 3 years (Supplementary Table 1), of which more than 50% were due to safety concerns, the valuation of public genomic medicine companies has fallen significantly (Supplementary Fig. 1). So, key questions for pharma companies and investors include how long will it take genomic medicines to realize their potential and how large will the associated markets be?
"With these questions in mind, we analysed the landscape of companies and their programmes for three types of genomic medicines: virally delivered gene therapies; regulatory oligonucleotides, such as antisense oligonucleotides and small interfering RNA (siRNA) therapies; and therapies based on genome editing technologies, such as CRISPR systems. Details of the definitions, datasets and analysis are provided in the Supplementary information.
"There are ~1,200 clinical trials of genomic medicines currently active (Fig. 1a). However, fewer than 20 genomic medicine products have been approved so far (Supplementary Table 2). Together, these treatments currently target <0.1% of the global population with genetic disorders.
"Among the three main genomic medicine platforms we analysed, the pipeline for gene therapies is the largest (532 programmes), but regulatory oligonucleotides are the basis for the greatest number of marketed products (11 products marketed for 13 indications) (Fig. 1a, Supplementary Table 2).
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If of interest, an AI summary of the article is here:
The landscape of genomic medicines is analyzed, revealing that while there are over 1,200 active clinical trials, fewer than 20 genomic medicine products have been approved. The pipeline for gene therapies is the largest, but regulatory oligonucleotides have the greatest number of marketed products. The focus is mainly on rare diseases, and the dominant delivery technologies are adeno-associated virus (AAV) vectors for gene therapies and genome editing.
The approval of genomic medicines is expected to occur in three waves over the next 20 years, with the first wave already underway and targeting rare eye disorders and monogenic diseases. The second wave will address a mix of non-rare and challenging rare disorders, while the third wave will target the most challenging rare diseases and non-rare diseases such as Alzheimer's and cancers. Challenges related to manufacturing, regulation, reimbursement, and pricing need to be addressed to fully realize the potential of genomic medicines.
- There are over 1,200 active clinical trials of genomic medicines, but fewer than 20 products have been approved so far.
- The pipeline for gene therapies is the largest, while regulatory oligonucleotides have the greatest number of marketed products.
- Over 50% of genomic medicine setbacks in the past three years were due to safety concerns.
- Gene therapies primarily use adeno-associated virus (AAV) vectors, while genome editing uses CRISPR systems.
- Rare diseases account for 53% of genomic medicine programs, and later-stage programs are more focused on rare diseases.
- The approval of genomic medicines is expected to occur in three waves over the next 20 years.
- The first wave targets rare eye disorders and monogenic diseases, the second wave addresses a mix of non-rare and challenging rare disorders, and the third wave focuses on the most challenging rare diseases and non-rare diseases like Alzheimer's and cancers.
- Challenges in manufacturing, regulation, reimbursement, and pricing need to be overcome for the full potential of genomic medicines to be realized.