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Category: Cell and Gene Therapy


Cell and Gene Therapy, Industry

Technology for In Vivo CAR T-cell Therapy Advances

February 2, 2023

Via: Biopharm International

There is no doubt that chimeric antigen receptor (CAR) T-cell therapies have had a tremendous positive impact on patients that have received them; however, autologous cell therapies produced in centralized manufacturing facilities have inherent limitations that inhibit widespread availability and […]


Cell and Gene Therapy, Industry, Mergers and Acquisitions

Andelyn Biosciences, Odylia Therapeutics Partner to Manufacture Gene Therapy for Vision Loss

February 1, 2023

Via: Contract Pharma

Andelyn Biosciences, a gene therapy contract development and manufacturing organization (CDMO), has partnered with Odylia Therapeutics, a nonprofit 501(c)(3) biotech focused on accelerating rare disease drug development, to manufacture a novel gene therapy (OT-004) to treat vision loss caused by […]


Cell and Gene Therapy, Industry

UniQure nabs another gene therapy for ALS

January 31, 2023

Via: Biopharma Dive

Apic Bio, a privately held developer of gene therapies for rare disorders, has agreed to sell the rights to one of its experimental ALS treatments to the biotechnology company UniQure in a deal announced Tuesday. Per deal terms, UniQure will […]


Cell and Gene Therapy, Clinical Trials, Industry, Research and Development

Be The Match BioTherapies Launches CRO Services for Cell and Gene Therapy

January 31, 2023

Via: Biopharm International

Be The Match BioTherapies, a provider of solutions for developing and commercializing cell and gene therapies, in conjunction with the Center for International Blood and Marrow Transplant Research (CIBMTR), is offering expanded clinical research organization (CRO) services. These services are […]


Cell and Gene Therapy, Industry

A startup launches with plans to open up a gene and cell therapy bottleneck

January 31, 2023

Via: Biopharma Dive

The cell and gene therapy field has a manufacturing problem. In recent years, research involving the complex medicines has accelerated. Multiple gene therapies are now approved in the U.S. to treat inherited diseases, while six cell-based treatments for blood cancers […]


Cell and Gene Therapy, Industry

Gilead’s CAR T-cell therapy recommended for use across the NHS

January 26, 2023

Via: Pharma Times

Treatment involves adult patients with relapsed or refractory diffuse large B-cell lymphoma Gilead Sciences has announced that the National Institute for Health and Care Excellence (NICE) has recommended Yescarta (axicabtagene ciloleucel) for routine commissioning on the NHS across England. It […]


Cell and Gene Therapy, Industry

Forge Biologics Receives PRIME Designation from EMA for Novel Gene Therapy

January 20, 2023

Via: Contract Pharma

Forge Biologics, a gene therapy-focused contract development and manufacturing organization (CDMO), said that the European Medicines Agency (EMA) has granted priority medicines (PRIME) designation to FBX-101, Forge’s lead adeno-associated virus (AAV) drug candidate and novel gene therapy for treating patients […]


Cell and Gene Therapy, FDA, Industry, Regulations

FDA lifts hold on Astellas’ Pompe gene therapy

January 20, 2023

Via: Pharmaphorum

The US regulator placed a hold on clinical testing of the gene therapy last June, shortly after a serious case of peripheral sensory neuropathy was reported in the phase 1/2 FORTIS trial in late-onset Pompe disease (LOPD). That came amid […]


Cell and Gene Therapy, Industry

EMA weighs new safety guidance for Novartis’ gene therapy Zolgensma

January 13, 2023

Via: Biopharma Dive

In its most severe form, spinal muscular atrophy is usually fatal before the age of two. Zolgensma, which was proven powerfully beneficial in clinical testing, won approval in Europe in 2020, one year after its clearance in the U.S. Testing […]


Cell and Gene Therapy, Industry

5 questions facing gene therapy in 2023

January 12, 2023

Via: Biopharma Dive

Sometime before the end of March, a person with the inherited blood disease beta thalassemia will receive an infusion containing hundreds of millions of their own stem cells. These cells will have just completed a round-trip journey to a drug […]


Cell and Gene Therapy, Industry

Nanotechnology may improve gene therapy for blindness

January 11, 2023

Via: World Pharma News

Using nanotechnology that enabled mRNA-based COVID-19 vaccines, a new approach to gene therapy may improve how physicians treat inherited forms of blindness. A collaborative team of researchers with Oregon Health & Science University and Oregon State University have developed an […]


Cell and Gene Therapy, Industry

Astellas licenses companion drug for Pompe gene therapy

January 10, 2023

Via: Pharmaphorum

The exclusive deal – which includes an upfront payment of $10 million and up to $340 million in milestone payments at the back end – covers the use of Selecta’s immunoglobulin G (IgG) protease drug IdeXork (Xork) alongside Astellas’ AT845, […]


Cell and Gene Therapy, Industry, Mergers and Acquisitions

Fujifilm Inks Cell Therapy Deal with Novo Nordisk

January 5, 2023

Via: Contract Pharma

Fujifilm Cellular Dynamics, a global developer and manufacturer of human induced pluripotent stem cells (iPSC), has entered an agreement to grant Novo Nordisk a non-exclusive right to use its iPSC platform for the development and commercialization of iPSC-derived cell therapies […]


Cell and Gene Therapy, Industry

Gene Therapy Breakthrough Helps Children Born Without Immune System

December 22, 2022

Via: Drugs.com

Ten children with an especially rare and hard-to-treat form of “bubble boy” disease are living normal lives after receiving a new gene therapy approach, researchers say. Experts said the findings are a major advance for children with the disease — […]


Cell and Gene Therapy, FDA, Industry, Regulations

FDA approves Adstiladrin as first gene therapy for NMIBC

December 19, 2022

Via: Pharmaphorum

Bladder cancer is one of the more common forms of cancer. According to the Centers for Disease Control and Prevention, about 57,000 men and 8,000 women are diagnosed with bladder cancer annually, and roughly 12,000 men and 4,700 women die […]


Cell and Gene Therapy, Industry

European regulators back hemophilia gene therapy

December 16, 2022

Via: Biopharma Dive

The agency that recommends what drugs should be approved in Europe has thrown its support behind five new ones, including a gene therapy for hemophilia, a combination treatment for liver cancer and a medicine for a rare disorder known as […]


Cell and Gene Therapy, FDA, Industry, Regulations

FDA approves first gene therapy for the treatment of high-risk, non-muscle-invasive bladder cancer

December 16, 2022

Via: World Pharma News

Today, the U.S. Food and Drug Administration approved Adstiladrin (nadofaragene firadenovec-vncg), a non-replicating (cannot multiply in human cells) adenoviral vector based gene therapy indicated for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) with […]


Cell and Gene Therapy, Industry

Gene Therapy Gel Offers New Hope Against Rare Blistering Disease

December 15, 2022

Via: Drugs.com

An experimental gene therapy that’s applied as a skin gel appears to heal wounds caused by a rare and severe genetic skin disease. Experts called the findings “remarkable,” and said they bring hope of a better quality of life to […]


Cell and Gene Therapy, Clinical Trials, Industry, Research and Development

Positive results for Amphera’s MesoPher cell therapy

December 12, 2022

Via: Pharma Times

Amphera – a company developing MesoPher cell therapy to treat cancer – has delivered positive results from its phase 2 Reactive trial and phase 2/3 DENIM trial. During the phase 2 Reactive study, patients with resected pancreatic cancer who had […]


Cell and Gene Therapy, Clinical Trials, Industry, Research and Development

Gilead buys into multiple myeloma cell therapy with Arcellx deal

December 9, 2022

Via: Biopharma Dive

Gilead placed a major bet on cell therapy with its $12 billion acquisition of Kite in 2017. To the frustration of some investors, the business took several years to ramp up. But sales have substantially grown in recent months. Gilead’s […]