Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
The cell and gene therapy sectors are growing exponentially, with new players emerging daily and much progress being made both in and out of the lab. CGTLive™’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.
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Catch up on CGTLive’s coverage of the American Heart Association’s (AHA) Scientific Sessions 2023, held November 10-13 in Philadelphia, Pennsylvania. Our coverage will continue throughout the week.
The FDA has approved Takeda Pharmaceuticals’ Adzynma (TAK-755) as a prophylactic or on demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP).
The FDA has granted investigational new drug (IND) clearance for an open-label, phase 2 trial of Kyverna Therapeutics’ KYV-101 chimeric antigen receptor T-cell (CAR-T) therapy in 10 patients with myasthenia gravis.
AviadoBio’s AVB-101, an investigational adeno-associated virus vector-based gene therapy intended to treat frontotemporal dementia associated with progranulin gene mutations, has received both clearance of its investigational new drug application and fast track designation from the FDA.
For World Cord Blood Day, recognized annually on November 15, the chairman and president of StemCyte discussed his thoughts on the importance of cord blood stem cells for the healthcare community.
As part of a new partnership with the California Institute for Regenerative Medicine (CIRM), contract development and manufacturing organization (CDMO) Avid Bioservices will cover activities such as manufacturing and process and analytical development for CIRM-funded programs that are developing gene therapies.
In a newly announced collaboration, Andelyn Biosciences will provide support in the form of manufacturing services, process optimization, and use of its platforms to the Ohio State University Gene Therapy Institute. The collaboration will focus on aiding the advancement of Ohio State University’s gene therapy programs for indications including Parkinson disease and amyotrophic lateral sclerosis.
The funding, which came in via private financing, will be put towards the ongoing development of VIPER-101, Vittoria’s autologous, dual population CD5-knockout CAR-T. "With a targeted focus on streamlined operations and an emphasis on cash efficiency, we plan to use this capital to advance our lead program, VIPER-101 - for the treatment of T-cell lymphoma - into the clinic in early 2024,” Nicholas Siciliano, PhD, the CEO of Vittoria, said in a statement.
Forge Biologics, which is both a CDMO focused on viral vectors and plasmids and a gene therapy developer with its own gene therapy product in clinical development for Krabbe disease, has agreed to be acquired by The Ajinomoto Group for $620 million in cash.
Arcellx and Kite, a Gilead Company, which previously established a strategic partnership late last year, have agreed to expand their collaborative activities in several key areas. Notably, Kite has exercised an option related to Arcellx’s multiple myeloma (MM) T-cell therapy ACLX-001 and the companies have agreed to expand their collaboration on Arcellx's CART-ddBCMA, initially also focused on MM, into lymphoma indications.
Under a new agreement, Novartis will pay $100 million to Legend upfront, with milestone payments and tiered royalties potentially coming down the pike, for exclusive global rights related to certain DLL3-targeted CAR-T therapies Legend is developing. Most notable is LB2102, for which Legend has received FDA clearance to initiate a phase 1 clinical trial in extensive stage small cell lung cancer and large cell neuroendocrine carcinoma. As part of the deal, Novartis has the right to implement its T-Charge CAR-T cell therapy platform into the manufacturing of the CAR-T therapies covered under the agreement.
Galapagos, which is developing CAR-T therapies for hematological cancers, has designed a decentralized manufacturing model intended to allow patients to receive autologous CAR T-cells at a median of 7 days following leukapheresis. Under the new agreement, Landmark will seek to implement this model during its manufacturing of clinical trial batches at its 44,000 square-foot development and manufacturing facility.