Category: Cell and Gene Therapy

November 28, 2023

Ogsiveo has been specifically approved for use in adults with progressing desmoid tumours who require systemic treatment. Each year, an estimated 1,650 people in the US are diagnosed with desmoid tumours, which may invade surrounding structures and organs, resulting in […]

November 28, 2023

The Food and Drug Adminsitration is investigating whether CAR-T cell therapies like Novartis’ Kymriah or Gilead’s Yescarta are linked to the risk of new blood cancers after receiving reports of so-called T cell malignancies in people who have received the […]

November 20, 2023

Last month, Bristol Myers delivered unwanted news, telling investors that sales of new drugs it’s counting on this decade will grow more slowly than expected. One of the chief culprits for that guidance shift was Abecma, sales of which dropped […]

November 17, 2023

Capivasertib has been approved as Truqap for use in combination with AZ’s oestrogen receptor antagonist Faslodex (fulvestrant) for adults with hormone receptor (HR)-positive and HER2-negative locally advanced or metastatic breast cancer, but only if they also have PIK3CA, AKT1, or […]

November 13, 2023

In signing Legend, Novartis has chosen a proven partner, as the biotech has already successfully developed the CAR-T drug Carvykti for the blood cancer multiple myeloma in partnership with Johnson & Johnson. These drugs involve taking patients’ T cells, re-engineering […]

November 7, 2023

The German biotech has signed a $1 billion-plus deal with China’s Biotheus, which includes $55 million upfront, to claim rights to a bispecific antibody for solid tumours in all markets outside Greater China. The drug – codenamed PM8002 – consists […]

November 7, 2023

AVB-101 will now benefit from the FDA’s fast track process, which is designed to improve the efficiency of product development and accelerate the review of treatments for serious conditions. FTD, a form of early-onset dementia, is the leading cause of […]

November 6, 2023

The results, which will be presented at the American Society of Hematology (ASH) Annual Meeting and Exposition, include long-term follow-up data from the company’s lentiviral vector (LVV) gene therapies in SCD patients who have a history of vaso-occlusive events (VOEs) […]

November 2, 2023

The potential therapy is the first to emerge from CRISPR’s and Vertex’s strategic partnership, which was expanded in 2021. Affecting more than 20 million people worldwide, SCD is a group of inherited red blood cell disorders which cause red blood […]

October 31, 2023

A decade ago, scientists outlined the gene editing potential of CRISPR, turning the vestiges of a bacterial immune system into one of the biotechnology industry’s most powerful tools. On Tuesday, a group of advisers to the Food and Drug Administration […]

October 30, 2023

Patients with sickle cell disease may soon have two new treatments to try. On Tuesday, a U.S. Food and Drug Administration advisory committee will weigh the merits of a new gene therapy for the painful, inherited condition, which typically strikes […]

October 25, 2023

The decision, which specifically applies to adults with relapsed or refractory (R/R) MDS with an IDH1 mutation, makes Tibsovo the first targeted therapy approved for this indication. The US regulator has also approved the use of a companion diagnostic, the […]

October 12, 2023

The FDA has shown caution with companies developing RNA treatments for neuromuscular conditions. Dyne Therapeutics, Sarepta Therapeutics, Avidity Biosciences and Entrada Therapeutics have all faced delays in their work amid partial or full clinical holds. PepGen appears to have resolved […]

October 10, 2023

The new facility is the latest investment from Bayer into cell and gene therapy. Bayer has been investing hundreds of millions of dollars into its Berkeley location, recently launching a Cell Culture Technology Center and cell therapy labs. Notably, the […]

October 5, 2023

Kyowa’s buyout of Orchard adds to a half dozen other gene therapy acquisitions over the past year. While the dealmaking is in some respects an encouraging sign, the sale prices have been modest and typically followed business setbacks for the […]

October 4, 2023

People with type 1 diabetes lack functional islet cells in their pancreas to produce the hormone insulin and must take daily insulin via injections or a continuous pump to compensate. But if new research pans out, some folks with type […]

September 25, 2023

On Sept. 21, 2023, the Novo Nordisk Foundation announced that it is investing up to DKK 950 million (€127 million, US$136 million) into a new cell therapy manufacturing facility in Lyngby, Denmark. The facility, which will be known as the […]

September 25, 2023

Food and Drug Administration scientists have “major concerns” with an application filed by biotechnology company BrainStorm Cell Therapeutics for approval of a personalized stem cell treatment for amyotrophic lateral sclerosis, documents posted online Monday show. According to the documents, FDA […]

September 21, 2023

On Sept. 20, 2023, Alexion, AstraZeneca’s rare disease group, completed the purchase and license agreement for a portfolio of preclinical rare disease gene therapy programs and enabling technologies from Pfizer. The acquisition cost AstraZeneca up to $1 billion, with the […]

September 21, 2023

Like many biotechnology companies, Seraxis has an ambitious aim. The company wants to transform the lives of millions of diabetics who must take insulin every day to control their blood sugar levels. Its approach: use a stem cell line generated […]