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ASGCT 2019: Remarkable Milestones and Achievements in Cell and Gene Therapy

am19-logo-150The American Society of Gene & Cell Therapy (ASGCT) held their 22nd annual meeting in Washington D.C. last week from April 29th through May 2nd bringing together a record number of over 3,400 attendees to share and discuss the remarkable work going on to advance this field. With 23 Scientific Symposia and 1000 abstracts presented through talks and poster sessions over the course of the conference, there was no shortage of knowledge to be shared.

Challenges and Advancements in the CAR-T Cell Therapy Race

CAR-T (chimeric antigen receptor) cell therapy was a hot topic with a great representation from the many companies racing to bring their products to the clinic for the targeted treatment of cancer. In honor of the founder of the ASGCT, George Stamatoyannopoulos, Dr. Michel Sadelain of Memorial Sloan Kettering Cancer Center gave a talk on the makings of these “living drugs” and how not only did one of the first come to be approved by the FDA (Yescarta) but also how we are working to continue to tackle the many challenges that still remain such as prolonging drug activity, curbing toxicity issues, and overall better engineer the CAR-T cell.

Other presentations were given highlighting the exciting advances in immunotherapy by groups like Cellectis whose allogeneic CAR-T cell platform (UCARTCS1A) targeting multiple myeloma can be large-scale manufactured, reducing the lag time for patients to receive treatment. For breast cancer, Minerva Biotechnologies shared results around the first in-human CAR-T for solid tumors that targets the MUC1 transmembrane and is expecting to enter the clinic by the end of the summer.

Remarkable Milestones in Gene Therapy

Gene therapy has had some remarkable milestone achievements recently and did not go unnoticed at the annual ASGCT meeting. AveXis, already a leader in the gene therapy field with their AVXS-101 for the treatment of spinal muscular atrophy type 1 (SMA1) set to receive regulatory approval this year, showed that they are making strides towards other targets as well. Brian Kaspar of AveXis presented data on the efficacy of AVXS-201 for the treatment of Rett Syndrome as well as extended lifespan with AVXS-301 for patients with ALS.

Another key group in the field of gene therapy, Spark Therapeutics, had quite the presence with talks on everything from how their product Luxturna became the first approved AAV product in the US to progress and challenges towards development of other gene therapies targeting severe life-threatening diseases. Even in the presentation of the top abstracts, gene therapy was a common denominator where researchers from Sanofi, UCSF, and the UCLA Medical Center all impressed with updates on current developmental endeavors.  

More Highlights from ASGCT 2019

AttieatWashingtonThe Exhibit Hall was a lively place filled with over 100 companies ready to share information around the latest and greatest products and services whether it be through demonstrations and conversations at their booths or through presentations in the Tools & Technologies Forums. These novel technologies, like that from Terumo BCT, with their cell expansion bioreactors and automated fill and finish systems or RoosterBio with their portfolio of stem cells and complementary cell culture media, are all designed to accelerate the development of cell and gene therapies.

Others shared technologies in the form of the actual cell based product such as bluebird bio who presented the process behind their gene addition and gene editing platforms or Sangamo Therapeutics who utilizes novel platform technologies in genome editing and cell therapy to treat an array of hematologic and metabolic diseases.

If all of this was not enough, there were hundreds of posters shared each night, networking receptions galore, and no shortage of opportunity for great conversations with the top minds in biotechnology today. Set in the heart of our nation’s capital, the 2019 ASGCT Annual Meeting was more than a success, setting the stage for an even better conference next year and with such rapid progress and advancements being made, we cannot wait to see what impressive developments will be presented in 2020.

Attie Pennybaker

Written by Attie Pennybaker

Atherly Pennybaker is a Product Applications Scientist with InVitria. At InVitria she enjoys leveraging her scientific background to help customers successfully transition to blood-free, chemically defined systems. Prior to InVitria, she received a Bachelor of Science in Chemical and Biological Engineering from Colorado State University. During her time there, she conducted undergraduate research in tissue engineering where she co-authored a paper on the osteoinductivity of an engineered biomimetic periosteum. She also worked on the design of a natural cell and tissue scaffold by means of electrospinning demineralized bone matrix.