Category: Cell and Gene Therapy

February 2, 2023

There is no doubt that chimeric antigen receptor (CAR) T-cell therapies have had a tremendous positive impact on patients that have received them; however, autologous cell therapies produced in centralized manufacturing facilities have inherent limitations that inhibit widespread availability and […]

February 1, 2023

Andelyn Biosciences, a gene therapy contract development and manufacturing organization (CDMO), has partnered with Odylia Therapeutics, a nonprofit 501(c)(3) biotech focused on accelerating rare disease drug development, to manufacture a novel gene therapy (OT-004) to treat vision loss caused by […]

January 31, 2023

Apic Bio, a privately held developer of gene therapies for rare disorders, has agreed to sell the rights to one of its experimental ALS treatments to the biotechnology company UniQure in a deal announced Tuesday. Per deal terms, UniQure will […]

January 31, 2023

Be The Match BioTherapies, a provider of solutions for developing and commercializing cell and gene therapies, in conjunction with the Center for International Blood and Marrow Transplant Research (CIBMTR), is offering expanded clinical research organization (CRO) services. These services are […]

January 31, 2023

The cell and gene therapy field has a manufacturing problem. In recent years, research involving the complex medicines has accelerated. Multiple gene therapies are now approved in the U.S. to treat inherited diseases, while six cell-based treatments for blood cancers […]

January 26, 2023

Treatment involves adult patients with relapsed or refractory diffuse large B-cell lymphoma Gilead Sciences has announced that the National Institute for Health and Care Excellence (NICE) has recommended Yescarta (axicabtagene ciloleucel) for routine commissioning on the NHS across England. It […]

January 20, 2023

Forge Biologics, a gene therapy-focused contract development and manufacturing organization (CDMO), said that the European Medicines Agency (EMA) has granted priority medicines (PRIME) designation to FBX-101, Forge’s lead adeno-associated virus (AAV) drug candidate and novel gene therapy for treating patients […]

January 20, 2023

The US regulator placed a hold on clinical testing of the gene therapy last June, shortly after a serious case of peripheral sensory neuropathy was reported in the phase 1/2 FORTIS trial in late-onset Pompe disease (LOPD). That came amid […]

January 13, 2023

In its most severe form, spinal muscular atrophy is usually fatal before the age of two. Zolgensma, which was proven powerfully beneficial in clinical testing, won approval in Europe in 2020, one year after its clearance in the U.S. Testing […]

January 12, 2023

Sometime before the end of March, a person with the inherited blood disease beta thalassemia will receive an infusion containing hundreds of millions of their own stem cells. These cells will have just completed a round-trip journey to a drug […]

January 11, 2023

Using nanotechnology that enabled mRNA-based COVID-19 vaccines, a new approach to gene therapy may improve how physicians treat inherited forms of blindness. A collaborative team of researchers with Oregon Health & Science University and Oregon State University have developed an […]

January 10, 2023

The exclusive deal – which includes an upfront payment of $10 million and up to $340 million in milestone payments at the back end – covers the use of Selecta’s immunoglobulin G (IgG) protease drug IdeXork (Xork) alongside Astellas’ AT845, […]

January 5, 2023

Fujifilm Cellular Dynamics, a global developer and manufacturer of human induced pluripotent stem cells (iPSC), has entered an agreement to grant Novo Nordisk a non-exclusive right to use its iPSC platform for the development and commercialization of iPSC-derived cell therapies […]

December 22, 2022

Ten children with an especially rare and hard-to-treat form of “bubble boy” disease are living normal lives after receiving a new gene therapy approach, researchers say. Experts said the findings are a major advance for children with the disease — […]

December 19, 2022

Bladder cancer is one of the more common forms of cancer. According to the Centers for Disease Control and Prevention, about 57,000 men and 8,000 women are diagnosed with bladder cancer annually, and roughly 12,000 men and 4,700 women die […]

December 16, 2022

The agency that recommends what drugs should be approved in Europe has thrown its support behind five new ones, including a gene therapy for hemophilia, a combination treatment for liver cancer and a medicine for a rare disorder known as […]

December 16, 2022

Today, the U.S. Food and Drug Administration approved Adstiladrin (nadofaragene firadenovec-vncg), a non-replicating (cannot multiply in human cells) adenoviral vector based gene therapy indicated for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) with […]

December 15, 2022

An experimental gene therapy that’s applied as a skin gel appears to heal wounds caused by a rare and severe genetic skin disease. Experts called the findings “remarkable,” and said they bring hope of a better quality of life to […]

December 12, 2022

Amphera – a company developing MesoPher cell therapy to treat cancer – has delivered positive results from its phase 2 Reactive trial and phase 2/3 DENIM trial. During the phase 2 Reactive study, patients with resected pancreatic cancer who had […]

December 9, 2022

Gilead placed a major bet on cell therapy with its $12 billion acquisition of Kite in 2017. To the frustration of some investors, the business took several years to ramp up. But sales have substantially grown in recent months. Gilead’s […]