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Tag: cell therapy


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An unusual drugmaker raises north of $100M for ecstasy-based PTSD therapy

January 5, 2024

Via: Biopharma Dive

A California-based company has raised tens of millions of dollars to support its mission of bringing an ecstasy-based therapy to patients with post-traumatic stress disorder. MAPS Public Benefit Corp. formed a decade ago as a subsidiary of the Multidisciplinary Association […]


Cell and Gene Therapy, Industry

Merck bets on Inspirna’s colorectal cancer drug ompenaclid

January 4, 2024

Via: Pharmaphorum

Germany’s Merck KGaA is paying $45 million upfront for rights to a potentially first-in-class drug for colorectal cancer (CRC) being developed by New York biotech Inspirna. The agreement gives Merck exclusive ex-US rights to ompenaclid – an oral inhibitor of […]


Cell and Gene Therapy, Industry

Pfizer wins Canadian OK for hemophilia treatment, its first gene therapy

January 3, 2024

Via: Biopharma Dive

Hemophilia and other blood disorders like sickle cell disease and beta thalassemia have drawn intense investment from genetic medicine developers. in December, the FDA approved two such treatments for sickle cell, clearing Vertex Pharmaceuticals’ gene editing drug Casgevy and Bluebird […]


Cell and Gene Therapy, Industry

Novartis deepens ties with Voyager via $100M gene therapy deal

January 2, 2024

Via: Biopharma Dive

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 […]


Cell and Gene Therapy, Industry

Replay Signs Exclusive Licensing Agreements with NIH and Miltenyi Biotec for Cell Therapy Development and Manufacturing

December 29, 2023

Via: Biopharm International

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 […]


Cell and Gene Therapy, Industry

Ultragenyx Picks Andelyn Biosciences to Manufacture Novel Gene Therapy for Sanfilippo Syndrome

December 27, 2023

Via: Biopharm International

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 […]


Mergers and Acquisitions

AZ makes another cell therapy play with $1.2bn Gracell buy

December 26, 2023

Via: Pharmaphorum

The agreement is the latest in a series of acquisitions and licensing deals aimed at bolstering AZ’s position in cell therapy, an area where it has lagged behind other companies like Novartis, Bristol-Myers Squibb, and Gilead Sciences, and also continues […]


FDA, Regulations

FDA establishes advisory committee for genetic metabolic disease treatments

December 18, 2023

Via: PMLiVE

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 […]


Cell and Gene Therapy, Industry

Stem Cell Therapy Boosts Quality of Life for People With Advanced Heart Failure

December 15, 2023

Via: Drugs.com

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 […]


Cell and Gene Therapy, Industry

FDA starts speedy review of Amgen’s lung cancer BiTE therapy

December 14, 2023

Via: Pharmaphorum

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 […]


Cell and Gene Therapy, Industry

FDA grants fast track designation to Candel’s immunotherapy in pancreatic cancer

December 13, 2023

Via: PMLiVE

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 […]


Cell and Gene Therapy, Industry

FDA approves two cell-based gene therapies for sickle cell disease

December 11, 2023

Via: PMLiVE

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 […]


Industry, Pharma

ASH23: Pharma branding, Editas’ high bar and clinical trial diversity

December 11, 2023

Via: Biopharma Dive

At the American Society of Hematology’s annual meeting, pharmaceutical branding is inescapable. Johnson & Johnson’s and GSK’s new logos are splashed across the side of phone charging stations. Sanofi, among others, let attendees know via hand sanitizer stands that it […]


Cell and Gene Therapy, Industry

CRISPR therapy for sickle cell approved by FDA in gene editing milestone

December 8, 2023

Via: Biopharma Dive

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. […]


Cell and Gene Therapy, Industry

Fujifilm to invest $200M in cell therapy production

December 6, 2023

Via: Biopharma Dive

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 […]


Cell and Gene Therapy, Industry

BMS chases MSD/Seagen with first-line bladder cancer filing

December 5, 2023

Via: Pharmaphorum

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 […]


Cell and Gene Therapy, Industry

CRISPR eyes autoimmune disease in revamp of cell therapy plans

December 5, 2023

Via: Biopharma Dive

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 […]


Cell and Gene Therapy, Industry

Lilly cancer drug cleared by FDA for expanded leukemia, lymphoma use

December 4, 2023

Via: Biopharma Dive

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 […]


Cell and Gene Therapy, Industry

AbbVie to expand solid tumour portfolio with $10bn ImmunoGen acquisition

December 1, 2023

Via: PMLiVE

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 […]


Cell and Gene Therapy, Industry

FDA to investigate risk of T-cell malignancy from CAR-T cell immunotherapies

November 30, 2023

Via: PMLiVE

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 […]