Tag: gene therapy

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 19, 2023

Checkpoint Therapeutics’ late entry into the PD-1/PD-L1 inhibitor category, cosibelimab, just got knocked back even further after it was turned down by the FDA as a treatment for skin cancer. A complete response letter (CRL) from the US regulator cites […]

December 18, 2023

The Genetic Metabolic Diseases Advisory Committee will advise the FDA on products used for the diagnosis, prevention and treatment of genetic metabolic diseases under the Division of Rare Diseases and Medical Genetics, established in 2020. Genetic metabolic diseases are conditions […]

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

A California company has asked the U.S. Food and Drug Administration to approve MDMA, the active ingredient in party drugs like molly and ecstasy, as a treatment for post-traumatic stress disorder (PTSD). When announcing the new drug application (NDA) filing […]

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 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 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

Flagship Pioneering is looking to raise $3 billion for its eighth venture fund, a sign the high-profile company creator continues to see investor interest in the biotechnology sector despite a turbulent market. The plans were outlined in a securities document […]

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

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 10, 2023

The first vaccine to prevent infection with the chikungunya virus was approved by the U.S. Food and Drug Administration on Thursday. The single-dose shot, known as Ixchiq, is approved for adults who are at increased risk of exposure to the […]

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 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 […]