Tag: cell therapy

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 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 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 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 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 7, 2023

Although diseases such as Ehlers-Danlos syndrome, Sanfilippo syndrome and Hemophilia are considered rare, their impact looms large. Rare diseases, defined as occurring in fewer than one in 2,000 individuals, affect more than 300 million people across the globe, and most […]

August 2, 2023

Former Biogen executive Michael Ehlers built Intergalactic with a vision to create a different type of gene therapy that wouldn’t need viruses as delivery systems. His team believed they could instead use electroporation, a method that uses electrical fields to […]

July 27, 2023

Kincell Bio, a technology-forward CDMO focused on cell therapies, has launched having received $36 million in new funding led by Kineticos Ventures. Kincell is a spinout of Inceptor Bio’s CMC, manufacturing and quality organizations formed to accelerate innovator research and […]

July 26, 2023

The report presents EU-level actions to address the challenges that were experienced during the COVID-19 pandemic and the outbreak of mpox disease in setting up adequately sized clinical trials across multiple member states. Organised in June 2023, with participation from […]

July 24, 2023

In January 2022, the Milken Institute published a report, “Cell and Gene Therapies: Looking Ahead to 2022,”1 describing the growth of the CGT industry in 2021. In the U.S., 7 new therapies were approved for commercialization, a similar number were […]

July 21, 2023

There hasn’t been much good news about biotech IPOs in the last year and a half, with the pace of new stock offerings dropping off substantially since a 2021 peak. But a recent flurry of activity over the last few […]

June 22, 2023

Today, the U.S. Food and Drug Administration approved Elevidys, the first gene therapy for the treatment of pediatric patients 4 through 5 years of age with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who do […]

June 20, 2023

Developing a new drug is a long, expensive process that comes with a high risk of failure, often because would-be medicines are unsafe or ineffective. For companies specializing in cell or gene therapies, an equally pressing concern is figuring out […]

June 16, 2023

Bispecific antibodies like Columvi are dual targeting, designed to bind to two cellular proteins, rather than one. In recent years, these types of drugs have won approval for use in multiple types of tumors, eye diseases and hemophilia. Development has […]