In June, Intellia Therapeutics thrilled the biopharma industry by announcing successful results from a clinical trial in which it used “in vivo” CRISPR/Cas9 genome editing to correct a rare genetic disease—meaning it successfully delivered the gene-editing technology directly into patients’ bodies and made the edit inside cells. The treatment outperformed the standard of care at reducing a key biomarker for the target disease.
In vivo gene editing is one of several advances in gene and cell therapy that are making their way into clinical trials. Another technology moving quickly through the biopharma pipeline is off-the-shelf CAR-T cell therapies for cancer that don’t have to be engineered from individual patients’ cells, like the current generation of CAR-T therapies do. And several companies are developing gene therapies that they hope to apply to more common diseases like diabetes and Alzheimer’s.
With these advances, however, comes challenges that will be difficult to overcome. Several of these treatments are designed as one-time therapies, raising questions about how they will be priced to balance patient and payer affordability with the need on the part of developers to recoup their investments. These sophisticated therapies will also require large-scale specialized manufacturing facilities that will be challenging to finance.
During this Fierce webinar, R&D experts from gene and cell therapy companies will come together to lay out the opportunities in the field and offer advice for overcoming the inevitable hurdles that will arise.
Chief Operating Officer
BaseCamp at ElevateBio
Michael has spent the last eighteen years in biotechnology leading and motivating process development and manufacturing teams to support programs from early development to late-stage clinical trials. Michael has been responsible for process development, analytics, cGMP manufacturing, CMC regulatory strategy, and process characterization and validation activities for multiple novel cell and gene therapy products. Currently, Michael is the Chief Operating Officer at ElevateBio, BaseCamp leading the operations for a broad portfolio of cell and gene therapy products and technologies.
Senior Vice President of Commercial Strategy and Operations
Ram Palanki, Pharm.D., joined REGENXBIO Management Team in August of 2018 as the Senior Vice President of Commercial Strategy and Operations. Dr. Palanki brings more than 15 years of experience in ophthalmology across pharmaceutical strategy and development, marketing, market access, sales, pricing, reimbursement, supply chain, distribution, business development, medical affairs and portfolio planning leadership.
Prior to joining REGENXBIO, he was Senior Vice President of Commercial for all of Americas at Santen Inc. Preceding Santen, he was Global Head of Marketing and Sales at Thrombogenics, Inc. where he led the commercial launch for Jetrea, a novel proteolytic enzyme therapy for symptomatic vitreomacular adhesion. Previously, Dr. Palanki served as Director of Global Marketing and Sales at NeoVista, Inc. and earlier in his career, held several positions at Genentech as part of the team that developed and launched Lucentis. Prior to Genentech, he worked at Eyetech Pharmaceuticals as part of the initial team submitting the New Drug Application for Macugen and at Novartis Pharmaceuticals in the New Product Commercialization group. Dr. Palanki earned his Pharm.D. at Albany College of Pharmacy in New York and completed his postdoctoral fellowship at Rutgers University in New Jersey.
VP Quality and Global Regulatory Affairs
Chaya is a specialist in regulatory and quality aspects both for gene and cell therapy as well as for complex molecules and biopharmaceuticals. She holds extensive experience in clinical development and operations and was involved in clinical and marketing regulatory submissions. Excellent track record of leading IND and CTA submissions, preparation of BLAs (MAA) and heading pre-approval inspections, while managing day-to-day regulatory and quality life-cycle aspects.
Chaya holds a BSc and MPH from the Hebrew University of Jerusalem.
Date: Thursday, December 2, 2021
Time: 11am ET / 8am PT
Duration: 1 Hour
Now Available On-Demand