ASGCT 2019 Insights: Cell and Gene Therapies Tackle 160+ Disorders
May 10, 2019
The American Society of Gene and Cell Therapy (ASGCT) annual meeting on Apr 28 – May 2 in Washington D.C. was superb. It brought together several thousands attendees. Prof. Steven Gray wittily noted that the number of attendees was similar to the number of orphan diseases. Hence, each of us should get to work. Arguably, the small size of the venue was conducive to additional bonding experience. People were gathering densely in front of extra monitors in hallways. However, some attendees did not appreciate packed sessions. But let’s look at the bright side – this community is growing by leaps and bounds. It is truly exciting.

The meeting featured approximately 1000 oral and poster presentations. Naturally, it was not humanly possible to attend all sessions of interest. Unless, one was Flash Gordon or had more than seven different devices, all streaming live. Hence, this article reviews the breadth of disease applications covered during the meeting. Note that the current summary excludes oncology.

Non-Oncology Disease Applications

Of 1000+ oral and poster presentations at ASGCT 2019, over 450 discussed specific disease applications of cell and gene therapy technologies. BioHeights estimated that 450+ presentations covered ~165 disorders (excluding oncology). Not surprisingly, neurology/ neuromuscular, metabolic, and hematology were the most common therapeutic areas. Top four most frequently tackled disorders included DMD, cystic fibrosis, Hemophilia A, and sickle cell disease. At the other end of the spectrum, approximately half of diseases appeared only once. The pie chart (Figure 1) shows a breakdown of 165 diseases by frequency of presentations.

Frequent Disease Applications Discussed at ASGCT

Figure 2 lists top 25 diseases that were discussed in 5 or more presentations. We made the best effort to separate unique diseases from a group they belong to. However, it was not possible in every research context. For example, sickle cell disease appears both separately and in the group of other hemoglobinopathies. Thus, when hemoglobinopathies were the focus of research, we did not separate sickle cell disease out.

*DMD: Duchenne muscular dystrophy; SMA: spinal muscular atrophy; A1AT: alpha-1 antitrypsyn; SCD: sickle cell disease; MPS: mucopolysaccharidosis; ALS: amyotrophic lateral sclerosis; MMA: methylmalonic acidemia; PKU: phenylketonuria; OTC: ornithine transcarbamylase deficiency; Rho RP; rhodopsin retinitis pigmentosa; nAMD: neovascular age-related macular degeneration.

Infrequent Disease Applications

Approximately 140 diseases were discussed in 4 or fewer talks or posters. As expected, the majority of those diseases are genetic and/or rare. Many of them have limited information on clinical phenotype or natural history. Often, scientists had to present the development of animal disease models from scratch, to lay the foundation for future therapeutic studies.

Undoubtedly, the level of scientific advancement achieved in the field of cell and gene therapy today is mind-boggling. For example, just a few years ago, many were skeptical about the ability (or even practicality) of gene therapy to target neuropathic pain or diseases caused by mutations in membrane proteins (e.g., CLN3). However, the 22nd ASGCT meeting was a testament to scientific creativity and ingenuity. More disorders became amenable to gene therapy approaches. Table 2 summarizes disorders and conditions that have been presented 2-4 times.

*dAMD: dry age-related macular degeneration; GAN: giant axonal neuropathy; GSD: glycogen storage disease; GvHD: graft-vs-host disease; hATTR: hereditary transthyretin-associated amyloidosis; HBV: hepatitis B; HSV: herpes simplex virus; IRDs: inherited retinal dystrophies; LGMD: limb-girdle muscular dystrophy; MLD: metachromatic leukodystrophy; MPS: mucopolysaccharidosis; SCID: severe combined immunodeficiency.

Other Diseases

The rest 78 diseases were presented at one talk or poster each.

We have analyzed and compiled the data from the 22nd ASGCT Annual Meeting. The Excel spreadsheet is available for download here. It contains disease/condition name, disease area, title of presentation, abstract number (where applicable), and affiliated organization. If you are interested in a more detailed analysis, please do not hesitate to email us at Significant discounts are available for current or former clients of BioHeights and for non-profit organizations and academic institutions. Email us for your discount code.

To find more interesting articles visit our Insights page.

Disclaimer: the content of this article was prepared based on the publicly available information. BioHeights is not responsible for the accuracy of that information. BioHeights is not responsible for any actions taken based on the content of this article or the corresponding downloadable disease list.
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