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Tag: gene therapy


FDA, Regulations

Bayer’s Nubeqa receives FDA approval to treat metastatic prostate cancer

August 9, 2022

Via: PMLiVE

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, […]


FDA, Regulations

AZ gets FDA nod for Enhertu in first HER2-low cancer

August 8, 2022

Via: Pharmaphorum

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 […]


Clinical Trials, Research and Development

Sosei Heptares banks $30m milestone in Neurocrine Bio alliance

August 5, 2022

Via: Pharmaphorum

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 […]


Cell and Gene Therapy, Industry, News

Sarepta says early filing for DMD gene therapy is back on

August 1, 2022

Via: Pharmaphorum

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 […]


Cell and Gene Therapy, Industry

AGC Biologics Supports Altheia Science’s Gene Therapy Programs

July 28, 2022

Via: Contract Pharma

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 […]


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 […]


Cell and Gene Therapy, Industry

VBL craters as ovarian cancer gene therapy fails phase 3 test

July 20, 2022

Via: Pharmaphorum

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 […]


Cell and Gene Therapy, Industry

Roche digs deeper into gene therapy for the eye

July 20, 2022

Via: Biopharma Dive

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, […]


Cell and Gene Therapy, Industry

Myocarditis case mars Sarepta DMD gene therapy readout

July 7, 2022

Via: Pharmaphorum

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 […]


News

Many Very Ill After Eating Daily Harvest Lentil Crumbles; FDA Investigating

July 1, 2022

Via: Drugs.com

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 […]


FDA, Regulations

FDA halts tests of Sanofi drug, acquired in $3.7B buyout, due to side effects

June 30, 2022

Via: Biopharma Dive

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 […]


Cell and Gene Therapy, Industry

J&J-backed gene therapy for the eye clears early study test

June 28, 2022

Via: Biopharma Dive

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 […]


Cell and Gene Therapy, Industry

Another Astellas gene therapy trial paused by FDA after side effect report

June 27, 2022

Via: Biopharma Dive

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 […]


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

BioMarin’s hemophilia gene therapy recommended for approval in Europe

June 24, 2022

Via: Biopharma Dive

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 […]


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 […]


Clinical Trials, Research and Development

Another amyloid miss in Alzheimer’s as Roche’s crenezumab fails trial

June 16, 2022

Via: Pharmaphorum

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 […]


Cell and Gene Therapy, Industry

Coave Therapeutics and ABL Enter Gene Therapy Collaboration

June 13, 2022

Via: Contract Pharma

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 […]


Cell and Gene Therapy, Industry, Manufacturing, Research and Development

Regenxbio Opens Gene Therapy Manufacturing Facility

June 9, 2022

Via: Contract Pharma

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. […]


Cell and Gene Therapy, Industry

BioMarin’s hemophilia A gene therapy further delayed amid new FDA questions

June 1, 2022

Via: FierceBiotech

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, […]