Tag: cell therapy

June 15, 2021

The parliament advocated for an increase in the number of NUB inquiries that can take place each year. NUB inquiries are used by hospitals to gain short term reimbursement for innovative treatment and diagnostic methods, supplementing the standard Diagnosis Related […]

June 15, 2021

The Phase III STAR study enrolled 169 adult males with a genetically confirmed diagnosis of choroideremia. It evaluated the safety and efficacy of a single subretinal injection of the investigational gene therapy. The primary endpoint was the proportion of patients […]

June 9, 2021

Beigene has quickly become a force in cancer drug development, earning approvals in the U.S. and China for its targeted therapy Brukinsa and immunotherapy tislelizumab. The biotech is developing a slate of small molecule and antibody drugs aimed at several […]

June 7, 2021

The approvals of Roche’s Luxturna (2017) for inherited retinal disease and Novartis’ Zolgensma (2019) for spinal muscular atrophy are indicative of a resurgence in the gene therapy field after a decline due to the death of a patient in 1999. […]

June 7, 2021

This includes the company’s lead pre-clinical ADC candidate IKS03, a potentially ‘best-in-class’ CD19-targeted asset for B-cell cancers. Iksuda will use the funds to support the progression of IKS03 into first-in-human Phase I clinical trials, and will also be used to […]

June 1, 2021

HS-001, Heartseed’s lead asset, is an investigational cell therapy using purified cardiomyocytes derived from induced pluripotent stem cells (iPSC). The therapy is currently being developed as a treatment for heart failure. Heartseed is already planning to launch a phase 1/2 […]

May 21, 2021

Data announced ahead of this year’s American Society of Clinical Oncology (ASCO) conference suggest that Gilead and CAR-T rivals Bristol-Myers Squibb and Novartis better watch their backs as Allogene announced plans for a pivotal study this year. Allogeneic or “off […]

May 14, 2021

Biogen’s acquisition of Nightstar signaled the biotech company’s commitment to gene therapy research, an investment it’s expanded upon with subsequent deals like a January collaboration with Germany’s ViGeneron and another earlier this week with Capsigen. The Nightstar deal bought Biogen […]

May 6, 2021

Two biotechs aim to broaden the reach of cancer immunotherapy by applying gene editing tools to an emerging form of cell therapy through a wide-ranging collaboration. The agreement, between CRISPR Therapeutics and Nkarta, includes three components. The biotechs will first […]

April 30, 2021

University of Tokyo researchers and colleagues at the RIKEN Research Institute have compiled a first-of-its-kind genetic database for autoimmune and autoinflammatory diseases. It’s hoped that the resource will offer up new insights into how immune disorders develop, and potentially aid […]

April 29, 2021

Gene therapy-focused CDMO Forge Biologics has closed on a $120 million Series B financing, saying the proceeds will accelerate its planned expansion of adeno-associated virus (AAV) manufacturing capabilities. The company operates a 175,000-square-foot cGMP facility in Columbus, OH, dedicated to […]

April 26, 2021

The IL-17A and IL-17F inhibitor hit all its objectives in the phase 2 trial, which has now been published in The Lancet, and showed that it was significantly more effective than placebo at clearing up skin lesions in patients with […]