Tag: cell therapy

July 25, 2022

Cell and gene therapies are taking the world by storm. With CRISPR gene-editing technology, the genome is no longer an untouchable sacred text. Instead, genomic DNA is actively manipulated in the laboratory and the clinic. This revolution is changing the […]

June 29, 2022

FDA unveiled its Action Plan for Rare Neurodegenerative Diseases including Amyotrophic Lateral Sclerosis (ALS) on June 23, 2022. The plan is a five-year strategy for improving and extending the lives of people living with rare neurodegenerative diseases through advancing the […]

June 27, 2022

The CHMP backed a conditional marketing authorisation for Roctavian (valoctocogene roxaparvovec or valrox) for severe haemophilia A in adults without inhibitors – antibodies that make standard Factor VIII replacement medicines less effective. Patients should also not antibodies to the adeno-associated […]

June 21, 2022

Vertex’s latest treatment for cystic fibrosis is a “triple combination” drug called Trikafta, which like others from Vertex boosts the activity of a protein that’s defective in people with the disease. It has quickly become a go-to treatment, entrenching Vertex’s […]

June 9, 2022

An experimental gene therapy could benefit young boys with a rare genetic disease, despite considerable concerns that treatment may cause cancer, a panel of Food and Drug Administration advisers unanimously agreed in a high-profile meeting Thursday. The therapy, an infusion […]

June 7, 2022

Though small in scope, Immatics and Editas’ deal adds to a flurry of recent activity involving treatments that harness gamma delta T cells, rare white blood cells with unique tumor-fighting capabilities. Unlike the T cells used in Novartis, Gilead and […]

June 6, 2022

Athersys’s research efforts are centered around a cell therapy platform it has been studying across a range of conditions, including stroke, respiratory distress and heart attack. In stroke, Athersys is working with Japan’s Healios, which tested Athersys’ cell therapy as […]

May 31, 2022

Despite a long history of difficulties in this area, FDA once again is taking steps to facilitate the import of less costly prescription drugs from Canada by states, Indian Tribes, and other entities. The Canadian government has objected to the […]

May 27, 2022

Most cancer immunotherapies primarily rely on T cells to neutralize tumors. But in two new studies, scientists at Dana-Farber Cancer Institute and Stanford University have described novel immuno-oncology approaches, showing that leveraging other components of the immune system may achieve […]

May 20, 2022

Bayer is pulling the plug on its $670 million CAR-T pact with Atara Biotherapeutics. Three months after a death paused a study of the lead candidate, the German drugmaker has decided to cut its ties to Atara’s two mesothelin-directed CAR-T […]

May 20, 2022

The FDA has sounded a warning about Foghorn Therapeutics’ blood cancer candidate FHD-286, putting a phase 1 clinical trial on a partial hold in response to the death of a patient with potential differentiation syndrome. FHD-286 is an enzymatic inhibitor […]

May 19, 2022

Syncona is continuing to bet big on gene therapy. While setbacks at other biotechs have taken the sheen off the modality, the investor is keeping the faith in SwanBio Therapeutics, stepping up to provide the lion’s share of a $56 […]

May 18, 2022

At a dedication of the new Jeffrey Leiden Center for Cell and Genetic Therapies in Boston’s Seaport, Vertex Pharmaceuticals unveiled plans to build another 344,000 square foot facility in the Seaport to support the company’s growth of its cell and […]

May 13, 2022

Editas Medicine has picked up more regulatory privileges for its ex vivo gene-edited medicine, securing FDA orphan-drug designation in beta thalassemia ahead of the start of a clinical trial in the indication. The candidate, EDIT-301, is in development as a […]

May 12, 2022

Cellipont Bioservices, a cell therapy contract development and manufacturing organization (CDMO), unveiled plans to expand its development and manufacturing facility at its Poway, CA location. The expansion will enable Cellipont to increase capacity for process development and Phase I/II programs. […]

May 9, 2022

The sun and shares are rising again at LogicBio after the FDA lifted a clinical hold on a genome editing therapy for children with a rare disorder. Shares of the Lexington, Massachusetts-based biotech were sent skyward by 60% as the […]

May 9, 2022

Aspen Neuroscience, a biotech developing the first autologous neuron replacement for Parkinson’s disease, is seeing double, as investors have flooded the company’s series B with $147.5 million following an initial $70 million round two years ago. The private neurodegenerative disease […]

May 5, 2022

On a quest to automate manual portions of the cell therapy production process, Multiply Labs is arming itself with two more pieces of industry-leading tech. The new kit comes on board a little less than a year after the robotics […]

May 4, 2022

Swiss biotechs saw record-breaking revenue and R&D investment in 2021, which the industry hopes will lead people to look beyond homegrown giants like Roche and Novartis. The new findings, outlined by the Swiss Biotech Association in its latest annual report […]

May 3, 2022

Zosano Pharma’s headaches are intensifying. With the FDA again knocking back its transdermal migraine patch, the biotech has suspended its M207 program and made more layoffs to preserve its dwindling cash reserves. The patch uses microneedles to deliver zolmitriptan, a […]