Tag: gene therapy

August 9, 2022

The decision was based on the ARASENS trial, which demonstrated that adding Nubeqa to standard androgen deprivation therapy (ADT) and docetaxel chemotherapy led to a statistically significant improvement in overall survival compared to ADT plus docetaxel and placebo. The results, […]

August 8, 2022

It has the potential to be a sizeable new indication for Enhertu (trastuzumab deruxtecan), a HER2-targeting antibody-drug conjugate, and central to AZ and Daiichi Sankyo’s hopes of unlocking multibillion-dollar sales for the drug. According to the FDA, out of the […]

August 5, 2022

The latest candidate to move into phase 2 is NBI-1117568, an oral, selective muscarinic M4 receptor agonist, which is the most advanced candidate in a $2.6 billion alliance with Neurocrine signed last year, and included an upfront payment of $100 […]

August 1, 2022

The US biotech – which already has three antisense drugs for DMD on the market – had said earlier it was planning to delay the application into 2023, so the new announcement marks an acceleration of its plans. Sarepta reported […]

July 28, 2022

AGC Biologics, a global Biopharmaceutical Contract Development and Manufacturing Organization (CDMO), entered a new partnership with Altheia Science, a gene therapy company pioneering cell and gene therapy strategies to treat autoimmune diseases and cancer. Under the alliance,Altheia Science aims to […]

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 […]

July 20, 2022

Shares in VBL Therapeutics have lost around 78% of their value in pre-market trading after the company reported a phase 3 trial of its lead gene therapy VB-111 for ovarian cancer failed a pivotal trial. In the OVAL study, VB-111 […]

July 20, 2022

Roche is putting more money into gene therapies for eye diseases, announcing a deal to work with University of Pittsburgh spinout Avista Therapeutics to develop better delivery tools for the complex treatments. Through the deal, Avista will develop new capsids, […]

July 7, 2022

Updated results from several trials of SRP-9001 (delandistrogene moxeparvovec) revealed a wealth of positives, including functional benefits in patients with the muscle-wasting disease after one to fours years of follow-up, pointing to a durable effect of the gene therapy. Sarepta’s […]

July 1, 2022

Fallout is mounting for the food delivery service Daily Harvest, which has voluntarily recalled a frozen food product suspected of sickening hundreds of customers in at least 26 states. The U.S. Food and Drug Administration is investigating. And an Oklahoma […]

June 30, 2022

Multiple sclerosis has a growing number of treatment options, including some newer pills that some patients prefer over older injectable or intravenous drugs that were once their only choices. Sanofi is in a race with Roche, Merck KGaA and Biogen […]

June 28, 2022

The first gene therapy for an inherited disease approved by the Food and Drug Administration was Luxturna, a treatment for a form of blindness caused by genetic mutations. The landmark clearance, coupled with the advantages of targeting the eye rather […]

June 27, 2022

Astellas paid $3 billion to acquire Audentes Therapeutics in December 2019 planning, like many of its peers, to capitalize on advances in gene therapy research. The deal came months after the FDA had approved the second gene therapy for an […]

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 24, 2022

The regulatory agency that recommends whether new drugs should be approved in Europe has thrown its support behind a gene therapy for the rare bleeding disorder hemophilia A, putting the potentially curative treatment one step closer to market. People with […]

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 16, 2022

The result isn’t a big surprise – Roche terminated two phase 3 trials of crenezumab in people with prodromal to mild sporadic Alzheimer’s in early 2019 after interim analyses found it would be futile to continue – but is another […]

June 13, 2022

Coave Therapeutics, a clinical-stage biotechnology company focused on developing life-changing gene therapies for CNS (Central Nervous System) and eye diseases, and ABL, a pure play contract development and manufacturing organization (CDMO) specialized in the development and manufacturing of viruses for […]

June 9, 2022

Regenxbio recently celebrated the opening of its new Manufacturing Innovation Center gene therapy manufacturing facility. “Launching operations at our Manufacturing Innovation Center is an important milestone in the evolution of REGENXBIO,” said Kenneth T. Mills, president and CEO of REGENXBIO. […]

June 1, 2022

BioMarin’s commercial aspirations for its hemophilia gene therapy remain on ice after the FDA lobbed additional questions for the company to address, pushing back an expected approval submission to September. BioMarin was vague on what the FDA is asking for, […]