Category: Cell and Gene 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 26, 2021

Ori Biotech, an innovator in cell and gene therapy manufacturing, and Vineti, a provider of digital enterprise platforms for cell and gene therapy supply chains, are partnering to produce a fully pre-integrated solution to modernize, fast-track, and streamline the manufacture […]

May 21, 2021

VectorY was chosen for its development of proprietary and partnered programs based on a novel adeno-associated viruses (AAV) platform for the treatment of muscular and neurodegenerative disorders. The company currently has a pipeline of vectorized antibodies, including two in pre-clinical […]

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 21, 2021

For a company that generated $13.4 billion last year, $5 million is hardly a make-or-break investment. So it goes with the $5 million gamble Biogen is taking on Ginkgo Bioworks’ cell programming platform and know-how. With an incentive-laden deal, Massachusetts-based […]

May 19, 2021

The 12-week results come from the first 11 participants enrolled in Sarepta’s Study SRP-9001-103 or ENDEAVOR study, which is being conducted in partnership with Swiss pharma company Roche. The study, which has so far enrolled 20 participants with DMD between […]

May 17, 2021

Scientists at University College London (UCL) have identified a new immunotherapy against hepatitis B virus (HBV), the world’s most common cause of liver cancer. The team’s study using immune cells isolated directly from patient liver and tumor tissue, showed that […]