Take a look at our most-read features from 2023 that explored the biggest current topics and concerns with gene and cell therapy development across treatment fields.
For all of 2023, the CGTLive™ team was following along the clinical development of targeted and novel engineered approaches to the treatment of patients with various medical disorders. These efforts included holding in-depth conversations with experts in the clinical care of these individuals, as well as in cell and gene therapy development, culminating in our coverage of each step of progress that the most exciting cellular and genetic treatments have made along the pipeline.
From major data publications and presentations to FDA decisions and major medical meetings, the team spent all year bringing the latest information to the website's front page.
While speaking to experts in the field or covering the biggest news updates, we identified some recurring themes and concerns that clinicians and researchers had within the field of cell and gene therapy, and wrote deeper dives into these topics than our everyday news coverage.
Some of these features have become our most-read pieces, because of the clinical impact, inventive mechanisms, or other topics they highlighted. Whatever the reason for the attention these stories got, their place here helps provide an understanding of the themes in cell and gene therapy over the course of 2023.
Here, we'll highlight some of the most-read features on CGTLive's cardiology page this year, including features from our new, biweekly Clinical Trials in Progress series. Click the buttons to read further into these stories.
October 20, 2023 — Prior to the approval of bluebird bio's lovo-cel (lovotibeglogene autotemcel), and CGTLive took a look at the therapy’s turbulent course of development and clinical trials evaluating the therapy for the treatment of sickle cell disease (SCD).
"I do support an approval; I think it's going to be very complicated, because we need to support SCD centers in delivering this product, as well as the product itself... I think it'll be a little bit complicated as we wander into this new territory of high-priced therapies."
– Julie Kanter, MD, of UAB
July 27, 2023 — Frontotemporal dementia (FTD) is the second most common form of dementia and typically affects patients under 65 years of age.1 FTD usually presents in either a behavioral or language variant, the latter of which can cause either a semantic type of dementia or a nonfluent aphasia type. There are currently no approved disease-modifying treatments specifically indicated for FTD
“It's devastating when they get this diagnosis. We have to tell them that there's no potential treatment options—we can only manage the symptomatic symptoms with behavioral modifications or speech and language therapy."
– Rebekah Ahmad, MD, PhD, of University of Sydney
June 16, 2023 — While the field of muscular dystrophy has achieved its first approval of a gene therapy, regulators have struggled to come to a consensus on the validity of microdystrophin as a surrogate end point for clinical benefit in pivotal clinical trials in the accelerated approval pathway.
“Several new therapies have been approved by the FDA for DMD using the presence of dystrophin as a surrogate end point. With this as established precedent, we expect additional clinical studies, including ongoing gene therapy clinical trials, to rely on dystrophin as a surrogate end point to predict clinical benefit and therefore potentially receive a marketing approval via accelerated approval from the FDA."
– Paul Melmeyer, MPP, of MDA
April 14, 2023 — For solid tumors, obstacles to bringing effective cell therapies to clinic have left patient populations waiting. Traditional cell therapy approaches, such as chimeric antigen receptor (CAR) T-cell therapies, have so far been unsuccessful in treating solid tumors because of unique challenges in these types of cancers, including tumor heterogeneity, targeting difficulties, and the tumor microenvironment. New treatment development tactics have begun to meet these challenges head on, and with creativity.
“For a long time, we thought that cell therapy was just going to be a hematologic malignancy thing. With the scientific advances in the last couple of years, I think it's very clear that solid tumor cell therapy is here. It's here to stay."
– Alison Betof Warner, MD, PhD, of Memorial Sloan Kettering
December 5, 2023 — Although the road has not been smooth, 2023 was witness to unprecedented progress in the treatment landscape of Duchenne muscular dystrophy (DMD), with the approval of Sarepta Therapeutics’ delandistrogene moxeparvovec (SRP-9001; Elevidys) earlier this summer. Despite the milestone approval, only a small percentage of patients with DMD currently stand to benefit from the therapy. There is much work to be done in the field to improve care for all patients with DMD as well as to answer questions about the gene therapy’s long-term durability, efficacy, and safety.
“We have many people younger than 4 and many people over the age of 5, that are still in need of treatment. So, I think while the landscape is really amazing, and building every single day... time matters to these people. Time means progression of disease."
– Pat Furlong, BSN, RN, of PPMD