Category: Cell and Gene Therapy

January 2, 2024

Novartis is a leading gene therapy developer. In 2019, its SMA treatment Zolgensma became one of the first genetic medicines cleared for use in the U.S., and so far has been the field’s most lucrative product. Since then, gene therapy […]

December 29, 2023

Replay, a genome writing company, announced that it has entered into separate agreements with the National Institutes of Health (NIH) and Miltenyi Biotec, a provider of products and services for biomedical research and cellular therapy, that will lead to the […]

December 27, 2023

On Dec. 18, 2023, Andelyn Biosciences, a contract development and manufacturing organization, announced that Ultragenyx has selected it to perform late-stage process performance qualification (PPQ) manufacturing of UX111, Ultragenyx’s investigational gene therapy under development for the potential treatment of Sanfilippo […]

December 15, 2023

Patients with advanced heart failure can benefit from stem cell therapy, a large, new clinical trial has found. Injections of stem cells programmed to heal damaged heart tissue wound up improving overall quality of life for heart failure patients, compared […]

December 14, 2023

Tarlatamab is under review as a treatment for adult patients with advanced SCLC who have disease progression on or after platinum-based chemotherapy, with the FDA due to deliver a decision on the marketing application by 12th June next year. The […]

December 13, 2023

The candidate, CAN-2409, has specifically been granted the designation for use alongside prodrug valacyclovir to improve overall survival in patients with pancreatic ductal adenocarcinoma (PDAC). CAN-2409 will now benefit from the FDA’s fast-track process, which is designed to improve the […]

December 11, 2023

Affecting approximately 100,000 people in the US, SCD is a life-long, genetic disease that causes red blood cells to take a distinct crescent shape, which can block blood vessels and affect the way oxygen is carried around the body. The […]

December 11, 2023

The results from the phase 3 APPEAR-C3G study showed that Fabhalta (iptacopan) met its primary objective of an improvement of protein in the urine (proteinuria) compared to placebo at six months, setting up filings for regulatory approval. Last week, targeted […]

December 8, 2023

The Food and Drug Administration on Friday approved a gene editing treatment for the blood disease sickle cell, following a few weeks behind regulators in the U.K. to clear the world’s first medicine built from the Nobel Prize-winning technology CRISPR. […]

December 6, 2023

PNH patients have an acquired mutation that causes them to produce red blood cells susceptible to premature destruction by the complement system, which can cause anaemia, thrombosis, fatigue and other symptoms that can impact quality of life. The disease has […]

December 6, 2023

Fujifilm said Tuesday it will invest $200 million in cell therapy manufacturing, earmarking the new funds for an area of drug research it expects to grow considerably in the coming years. The investments will go to two subsidiaries focused on […]

December 5, 2023

The filing is based on the CheckMate-901 trial, which showed that adding PD-1 inhibitor Opdivo (nivolumab) to standard first-line cisplatin and gemcitabine chemotherapy improved survival in cisplatin-eligible patients with untreated, unresectable, or metastatic UC, the most common form of bladder […]

December 5, 2023

CRISPR Therapeutics achieved a major scientific milestone last month with the U.K. approval of Casgevy, the first in the world for a CRISPR medicine. Wall Street analysts are expecting a similar outcome with U.S. regulators. Yet the sales outlook for […]

December 4, 2023

Lilly’s oncology division is sometimes overshadowed by its metabolic medicine and neurology businesses, but cancer drugs were the second biggest contributor to the company’s $25 billion revenue through the first nine months of the year. Long-lived drugs like Alimta and […]

December 1, 2023

The definitive agreement grants AbbVie access to ImmunoGen’s antibody-drug conjugate (ADC) Elahere (mirvetuximab soravtansine-gynx), which received accelerated approval from the US Food and Drug Administration last year to treat platinum-resistant ovarian cancer. ADCs are a new class of cancer therapies […]

November 30, 2023

Following reports from clinical trials and post-marketing adverse event data sources, the wide probe is directed at patients who received treatment with all currently approved BCMA- or CD19-directed autologous CAR-T cell immunotherapies. CAR-T-cell therapy is a type of immunotherapy that […]

November 28, 2023

The probe is directed at all CAR-Ts currently on the US market, including those that target CD19, as well as BCMA, which are used to treat a range of haematological malignancies, such as various forms of non-Hodgkin’s lymphoma, acute lymphoblastic […]

November 28, 2023

Results from the trial show potential for developing an advanced cell therapy treatment for the condition. Affecting around two million people globally, MS is a neurodegenerative condition that affects the brain and spinal cord. Around two-thirds of people with MS […]

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 […]