Category: Cell and Gene Therapy

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 […]

September 20, 2023

Taysha is now retrenching and focusing on another experimental drug, TSHA-102, for a rare neurological disorder called Rett Syndrome. The company now expects to reduce operating expenses enough to extend its cash runway into the fourth quarter of 2025. While […]

September 19, 2023

In recent years, cancer researchers have hailed the arrival of chimeric antigen receptor T cell (CAR T) therapy, which has delivered promising results, transforming the fight against various forms of cancer. The process involves modifying patients’ T-cells to target cancer […]

September 14, 2023

AlveoGene sprung out of the work of the U.K. Respiratory Gene Therapy Consortium, a group originally founded in 2001 to find a gene therapy for cystic fibrosis. In the process of developing a viral vector to deliver the cystic fibrosis […]

September 11, 2023

Novartis has stopped work on a gene therapy candidate it acquired a little under two years ago, according to an investor in the company the Swiss drugmaker bought. The decision to discontinue research follows a recommendation from a data monitoring […]

September 5, 2023

Success in the clinic would boost the RNA editing field as well as Wave. Broadly speaking, the technology is designed to edit specific sites in an RNA transcript without making permanent changes to a person’s genome. It’s an approach that’s […]

September 5, 2023

The funding will boost the University of Nottingham spinout’s development of bone regeneration treatment TherageniX, a company developing a dry powder gene therapy for bone graft augmentation, and the institution from which it emerged, the University of Nottingham, have been […]

August 31, 2023

Jeff Ajer, BioMarin’s executive vice president and chief commercial officer, said the announcement “represents an important milestone” for the haemophilia community, as well as for patients and physicians around the world seeking access to the therapy. Haemophilia treatment centres in […]

August 30, 2023

The trial results were presented at the International Congress of Parkinson’s Disease and Movement Disorders in Copenhagen, Denmark. Parkinson’s disease is the fastest-growing neurodegenerative disease in the world and causes parts of the brain to become progressively damaged over many […]

August 28, 2023

Estimates hold that around half a million people in the U.S. have Parkinson’s, though, due to underdiagnosis or misdiagnosis, some believe the true number is much higher. The disease causes nerve cells in the part of the brain that controls […]

August 24, 2023

Royalty Pharma, a company specializing in acquiring pharmaceutical royalties, and Ferring Pharmaceuticals, a biopharmaceutical company with a new gene therapy expected to hit the market, announced that Royalty Pharma had acquired a synthetic royalty on US net sales of Ferring’s […]

August 21, 2023

After the success of cancer immunotherapies like Keytruda and Opdivo, drugmakers went on a hunt to find new ways to defeat cancer by turning the immune system onto tumor cells. One such pathway was CD47, a cell protein that sends […]

August 16, 2023

The FDA’s decision to skip an advisory panel may indicate that agency reviewers are more unified in their views of the Bluebird therapy. Or it could simply indicate a higher comfort level with the type of treatment Bluebird is developing; […]

August 15, 2023

CAR-T therapies are engineered from patients’ own T cells to attack cancer. The process requires the cells to be withdrawn from patients’ bodies and manufactured into the treatment, a time lag that makes recipients vulnerable to disease progression in the […]

August 14, 2023

As with other types of cancers, researchers have sought ways to turn the immune system against multiple myeloma. Their efforts have borne fruit with engineered T cell therapies like Bristol Myers Squibb and 2Seventy Bio’s Abecma and J&J and Legend […]

August 9, 2023

In the ongoing search for a cure for Alzheimer’s disease, a burgeoning branch of medicine is bringing new hope. Stem cell therapies are already being used to treat various cancers and disorders of the blood and immune system. In a […]

August 8, 2023

Under the terms of the agreement, Astellas will invest a total of $50m, including $25m to acquire 8,333,333 in shares of the common stock of Poseida, as well as a one-time $25m payment for a right of exclusive negotiation and […]