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Tag: cell therapy


Cell and Gene Therapy, Industry

Next-level quality control in cell and gene therapy

July 25, 2022

Via: Biopharma Dive

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 […]


FDA, Regulations

FDA Releases Action Plan for Rare Neurodegenerative Diseases

June 29, 2022

Via: Biopharm International

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 […]


Cell and Gene Therapy, Industry

BioMarin gene therapy for haemophilia on course for EU approval

June 27, 2022

Via: Pharmaphorum

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 […]


Cell and Gene Therapy, Industry

A biotech startup launches with $38M to develop a cystic fibrosis gene therapy

June 21, 2022

Via: Biopharma Dive

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 […]


Cell and Gene Therapy, Industry

FDA panel backs Bluebird gene therapy despite safety risks

June 9, 2022

Via: Biopharma Dive

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 […]


Cell and Gene Therapy, Industry

Immatics and Editas join up to bring CRISPR to ‘gamma delta’ cell therapy

June 7, 2022

Via: Biopharma Dive

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 […]


Biotech, Industry

Biotech Athersys to lay off 70% of staff in cost-cutting effort

June 6, 2022

Via: Biopharma Dive

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 […]


Cell and Gene Therapy, Industry

Evotec Adds Cell Therapy Mfg. Facility with Rigenerand Acquisition

May 31, 2022

Via: Contract Pharma

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 […]


Cell and Gene Therapy, Industry

Novel cancer vaccines by Dana-Farber and Stanford look beyond T cells to better fight tumors

May 27, 2022

Via: FierceBiotech

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 […]


Cell and Gene Therapy, Industry

Bayer pulls plug on $670M CAR-T cell therapy pact with Atara in wake of patient death

May 20, 2022

Via: FierceBiotech

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 […]


Clinical Trials, Research and Development

Death triggers FDA hold on Foghorn blood cancer clinical trial, shrouding timing of data drop

May 20, 2022

Via: FierceBiotech

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 […]


Cell and Gene Therapy, Industry

With lead gene therapy nearing the clinic, SwanBio leans heavily on Syncona to close $56M series B

May 19, 2022

Via: FierceBiotech

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 […]


Cell and Gene Therapy, Industry

Vertex Expands Cell and Gene Therapy Footprint

May 18, 2022

Via: Contract Pharma

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 […]


Cell and Gene Therapy, Industry

FDA tags Editas’ thalassemia cell therapy an orphan drug as it rounds final bend in race to clinic

May 13, 2022

Via: FierceBiotech

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 […]


Cell and Gene Therapy, Industry

Cellipont Bioservices Expands Cell Therapy Facility

May 12, 2022

Via: Contract Pharma

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. […]


Cell and Gene Therapy, Industry

LogicBio’s sun and shares rise again after FDA releases clinical hold on pediatric genome editing therapy

May 9, 2022

Via: FierceBiotech

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 […]


Cell and Gene Therapy, Industry

Investors take root in Aspen Neuroscience’s customized cell therapy as $147.5M round closes

May 9, 2022

Via: FierceBiotech

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 […]


Cell and Gene Therapy, Industry

Thermo Fisher, Charles River join robotics outfit Multiply Labs’ quest to automate cell therapy production

May 5, 2022

Via: FiercePharma

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 […]


Biotech, Industry

Record-breaking 2021 revenue shows there’s more to Swiss biotech sector than Roche, Novartis

May 4, 2022

Via: FierceBiotech

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 […]


FDA, Regulations

No go for Zosano as FDA knockback triggers suspension of transdermal migraine patch program

May 3, 2022

Via: FiercePharma

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 […]