This year we reached out to numerous experts outside the context of the conference cycle to seek their insight on company announcements, pipeline therapies, and disease awareness days.
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. Although our team covered updates from many major medical society meetings this year, we also reached out to experts outside the context of the conference cycle to seek their insight on recent company announcements, to get their perspective on a disease awareness day, or to simply learn more about a particular gene or cell therapy in the pipeline.
Here, we'll highlight some of the most-watched interviews from CGTLive's Videos page this year that were not directly related to ongoing medical conferences. Click the buttons to watch.
The current standard of care for stage 4 non–small cell lung cancer (NSCLC) is a combination treatment consisting of repeated dosing with carboplatin, pemetrexed, and pembrolizumab. Unfortunately, the progression-free survival (PFS) rate at 5 years for patients in this population is only 7%.
"So, if we can get memory T-cells specific against tumor antigens that are able to continually patrol and destroy tumor cells as they arise over the course of decades, we have a better chance of getting a real meaningful regression of cancers.”
— Ben Creelan, MD of Moffitt Cancer Center
IDCT (rebonuputemcel; DiscGenics) yielded durable improvements in low back pain, function, quality of life, and pain medication usage by 12 weeks in patients with lumbar degenerative disc disease (DDD) in 2-year data from the phase 1/2 DGX-A01 study (NCT03347708).
“I have never seen anything regrow a disk, but I've seen DiscGenics’ cellular therapy do it and we were able to show that in our data also. The high dose group showed dramatic improvement, visibly and from a mathematical computation, showing that disk volume increased. These are signs that we're actually regenerating the spine, which should excite everyone.”
— Matthew Gornet, MD, of The Orthopedic Center of St. Louis
Historically, the standard of care for Rett syndrome has largely consisted of physical therapy and speech therapy intended to ameliorate symptoms, as no FDA-approved specifically-targeted drugs existed for the condition. Earlier this year, however, Acadia Pharmaceuticals’ trofinetide (marketed as Daybue) was approved by the FDA for the treatment of Rett syndrome.
“TSHA-102 is a gene therapy given intrathecally into the lumbar area between L4 and L5. It's a simple procedure done by lumbar puncture. That allows it to be done in multiple sites in many parts of the country, even in an outpatient setting, inpatient setting, or in a hospital, etc. Ease of use is paramount here...”
— Sukumar Nagendran, MD, of Taysha Gene Therapies
September is Duchenne Action and Muscular Dystrophy Awareness Month, and CGTLive is speaking with clinicians and advocacy groups in the space to learn more about the progress in the field to date and unmet needs that remain. While 2023 has seen lots of progress in the treatment landscape of the field, including the summer approval of delandistrogene moxeparvovec (marketed as Elevidys by Sarepta), the field’s first gene therapy, more work always remains to be done.
“Some of the concerns that we have today are with these very high dose serious adverse events, that's one of the challenges with Duchenne. When you think that the body is comprised of 40 to 60% muscle – the amount of AAV that we need to put in the body in order to target all the different muscle groups is extremely high."
— Michael Kelly, PhD, of CureDuchenne
Epidermolysis bullosa (EB), a collection of rare genetic connective tissue disorders, remains highly difficult to manage for patients: until very recently, treatment for these disorders was mostly limited to palliative care. Fortunately, this is already changing, with the recent approval of Krystal Biotech’s beremagene geperpavec (B-VEC, marketed as Vyjuvek), a topical and redosable gene therapy for dystrophic epidermolysis bullosa (DEB), in May 2023, and a number of still-investigational advanced therapies on the near-term horizon.
“I think when we are discussing being able to treat this disease effectively, we really need to look at a holistic approach to it... I can see in the near future where a patient is actually using more than one cell and gene therapy.”
— Brett Kopelan, MA, of debra of America