Kite Pharma, Inc.

– U.S. IND clearance follows recent approval of CTA for EPI-321 in facioscapulohumeral muscular dystrophy (FSHD) in New Zealand – Global Phase 1/2 clinical trial expected to commence in 2025 SOUTH SAN FRANCISCO, CA, USA I April 03, 2025 I Epicrispr Biotechnologies , a biotechnology company focused on developing curative therapies, today announced that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for EPI-321, a first-in-class epigenetic therapy for the treatment of facioscapulohumeral muscular dystrophy (FSHD), a genetic neuromuscular disease. IND clearance in the US is part of a global development strategy for EPI-321 and follows the recent approval of its clinical trial application by New Zealand’s Medsafe. The company plans to initiate a global Phase 1/2 clinical trial in 2025. EPI-321 is an investigational one-time gene-modulating therapy designed to silence aberrant expression of DUX4, a gene that is incorrectly activated in FSHD and leads to progressive muscle degeneration. Delivered systemically via a clinically validated AAV vector, EPI-321 has demonstrated robust suppression of DUX4 expression and protection of muscle tissue in preclinical models. EPI-321 has received FDA Fast Track, Rare Pediatric Disease, and Orphan Drug designations. “For too long, individuals with FSHD have faced a progressive disease with no treatment options,” said Amber Salzman, Ph.D., Chief Executive Officer, Epicrispr Biotechnologies. “EPI-321 is the first therapeutic candidate designed to silence DUX4 through epigenetic reprogramming, offering a potentially transformative approach to halting disease progression in FSHD. We look forward to initiating our study in the US and bringing EPI-321 to patients who urgently need therapies.” “FDA clearance of EPI-321 is a significant milestone for the FSHD community,” said Russell Butterfield, M.D., Ph.D., Associate Professor in the Department of Neurology and Pediatrics, University of Utah. “Patients and families have long lacked options for this progressive neuromuscular disorder. EPI-321’s innovative approach targeting the root cause of FSHD offers new hope for treatment. We are optimistic about the upcoming U.S. clinical trial and the potential benefits this therapy could bring to individuals living with FSHD.” About Epicrispr Biotechnologies Epicrispr Biotechnologies is a biotechnology company pioneering gene-modulating therapies, leading with treatments for neuromuscular diseases. The company’s proprietary Gene Expression Modulation System (GEMS) enables precise, durable control of gene expression, unlocking first-in-class treatments for previously untreatable conditions. Epicrispr’s lead program, EPI-321 is in clinical trials for FSHD, and the company is advancing additional gene-modulating therapies. Epicrispr also has a research collaboration with Kite Pharma to develop next-generation CAR T-cell therapies. Learn more at www.epicrispr.com or follow us on LinkedIn . SOURCE: Epicrispr Biotechnologies

To qualify for advanced manufacturing technology designation from the FDA, a company must present a manufacturing approach that incorporates a new technology or uses an established technology in a novel way to substantially improve the production process for a drug. A little over a year after winning manufacturing certification for its cell therapy factory-in-a-box, South San Francisco’s Cellares has passed another major production milestone.Cellares’ automated cell therapy production platform, known as the Cell Shuttle, has received a coveted advanced manufacturing technology (AMT) designation from the FDA’s Center for Biologics Evaluation and Research, the company announced this week. Cellares—which pitches itself as the industry’s first integrated development and manufacturing organization—says the designation should bolster the adoption of innovative technologies, like the Cell Shuttle, that have the potential to boost the efficiency, quality and scalability of cell therapies.The manufacturing platform itself, which earned a current good manufacturing practice (cGMP) seal of approval last March, is about as big as a truck and combines all the technologies needed to complete an end-to-end cell therapy production run in a single piece of equipment, Cellares has explained.After initially floating a framework for the classification in late 2023, the FDA in December released its final guidance on the AMT designation program.To qualify for AMT designation, a company—be it a drugmaker, contract manufacturer or technology developer—must present a manufacturing approach that incorporates a new technology or uses an established technology in a novel way to substantially improve the production process for a drug while maintaining or enhancing its qualities, according to the FDA’s guidance.The FDA’s criteria for inclusion in the AMT club also covers technologies that cut development timelines or boost supplies of drugs that are critical or in shortage.“The AMT designation for our Cell Shuttle is a testament to its potential to transform patient outcomes by delivering scalable and cost-effective cell therapy manufacturing to small, early-stage biotechs and large pharma companies with commercialized cell therapies,” Fabian Gerlinghaus, Cellares’ CEO and co-founder, said in a statement about the new FDA classification. AMT designations have many benefits, according to the FDA, including the potential to provide greater quality assurance, help the industry “more efficiently” meet regulatory standards for commercial manufacturing and strengthen “regulatory predictability” for products using the platforms, the agency explained in its guidance document.Cellares, six years into its run, has attracted some impressive partners eager to get their hands on the startup’s automated approach to cell therapy. Production of cell therapies has long been a limiting factor for the field, with scalability, labor intensity and cost frequently cited as pain points by manufacturers.Last April, Bristol Myers Squibb expanded an ongoing partnership with Cellares through a $380 million deal to reserve manufacturing space for CAR-T therapies. The deal, which includes both upfront and future milestone payments, grants BMS access to an undisclosed number of Cellares’ Cell Shuttles in the U.S., Europe and Japan, the companies said at the time.BMS, for its part, said the collaboration will boost capacity for its established CAR-T treatments Breyanzi and Abecma plus future cell therapy candidates. Just a few months later, in June, Gilead Sciences’ Kite Pharma unit linked up with Cellares in a proof-of-concept evaluation of the Cell Shuttle to determine whether the platform could be a viable option for Kite. The path is similar to that which BMS followed before stepping up its Cellares commitment with a full-on manufacturing pact last year. 

A New York City drug developer led by well-known executives and backed by some of the top names in biotech investing is shutting down, a blow to a company that was prepping for a pivotal trial in colorectal cancer. Inspirna is “winding down” after “10 years as a fantastic biotech company,” CEO Oz Azam wrote in a LinkedIn post on Monday morning. The cell and gene therapy veteran joined Inspirna in 2023 after leading Empyrean Neuroscience and Tmunity Therapeutics. Inspirna’s team decided to close the company after a Phase 2 trial of its oral small molecule, called ompenaclid, “did not read out positively” in KRAS colorectal cancer, according to a source familiar with the decision, who agreed to speak on the condition of anonymity. Merck KGaA paid $45 million upfront last year for the ex-US rights to the SLC6A8 inhibitor and had the option to co-develop and co-promote it in the US. The companies were also working on follow-up SLC6A8 compounds. Merck KGaA will “not pursue further development of ompenaclid” and will not exercise its option in the US, a spokesperson for the drugmaker told Endpoints News via email. Azam did not immediately respond to an inquiry from Endpoints. Inspirna is at least the seventh biotech to shutter so far in 2025, following last week’s news about Lyndra Therapeutics . Inspirna was putting together a global pivotal study of ompenaclid for patients with certain forms of colorectal cancer, but the trial “requires additional funding and is dependent on further FDA feedback,” according to the company’s first-quarter presentation . Inspirna last disclosed a $50 million Series D in July 2022. Its backers included blue-chip investors like Vivo Capital, Sands Capital, Novo Holdings, and Sofinnova Partners. The financing was expected to keep the R&D engine humming until the second half of 2024, a spokesperson told Endpoints at the time of the Series D. Inspirna also had a small molecule called abequolixron in its pipeline. The LXR-beta selective agonist was being tested in Phase 1b/2 for certain forms of lung cancer. GSK discovered the asset and originally intended for it to treat cardiovascular disease before licensing it out to Inspirna. Inspirna was formed more than a decade ago as Rgenix from the Tavazoie Lab at The Rockefeller University. The biotech’s platform focused on microRNA, or miRNA, dysregulation, which is thought to contribute to the development of cancer and its progression. Dieter Weinand, a former CEO of Bayer’s pharma division, chaired Inspirna’s board.