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Tag: gene therapy


Cell and Gene Therapy, Industry

Mainstreaming cell and gene therapy – Realizing its potential

November 28, 2022

Via: Biopharma Dive

Brief overview of the CGT landscape A useful starting point is to understand the different types of CGTs. The following are the definition of the three types: Cell therapies: involves the transfer of intact, live cells into a patient to […]


Cell and Gene Therapy, FDA, Industry, Regulations

uniQure, CSL claim first okay for haemophilia B gene therapy

November 23, 2022

Via: Pharmaphorum

The adeno-associated virus (AAV) vector-based therapy is given as a one-shot intravenous infusion, delivering a gene for Factor IX, a clotting factor that is deficient in haemophilia B. In trials, it cut the annualised bleed rate for patients, as well […]


Clinical Trials, Research and Development

X Chromosome Inactivation Surprisingly Found in Diverse Cancers in Men

November 10, 2022

Via: GEN

Cancer cells acquire genetic changes that allow them to grow and proliferate unchecked. Researchers at the Dana-Faber Cancer Institute and colleagues have now found another difference between cancer cells and normal cells. The team analyzed public datasets comprising thousands of […]


Cell and Gene Therapy, Industry

Replay launches gene therapy spinout Eudora, putting ‘hub-and-spoke’ plans to work

October 31, 2022

Via: Biopharma Dive

Replay, a biotechnology startup built around several gene therapy technologies, has launched the first of several planned spinouts, revealing on Monday a company named Eudora. Replay is only three months from its own debut, having emerged in July with $55 […]


Cell and Gene Therapy, Industry, Mergers and Acquisitions

Astellas to support development of Taysha’s AAV-based gene therapy programmes

October 26, 2022

Via: PMLiVE

Under the terms of the agreement, the Japanese firm will invest a total of $50m to acquire 15% of the outstanding common stock of Taysha and to receive an exclusive option to license two of Taysha’s clinical stage programmes: TSHA-102 […]


Cell and Gene Therapy, Industry, Mergers and Acquisitions

Astellas makes another gene therapy play, takes stake in Taysha

October 25, 2022

Via: Pharmaphorum

The Japanese firm has agreed to make a $50 million investment in Dallas-based Taysha in exchange for a 15% stake in the company, plus exclusive options to license two clinical-stage, single-gene therapies for rare genetic diseases. The option covers TSHA-102, […]


Mergers and Acquisitions

Gamma Biosciences, Lonza Enter Cell and Gene Therapy Alliance

October 14, 2022

Via: Contract Pharma

Gamma Biosciences, a provider of tools and technologies for cell and gene therapy manufacturing, through their BioMagnetic Solutions business, entered a strategic collaboration with Lonza. Under the multi-year agreement, BioMagnetic Solutions will supply its research and cGMP manufacturing grade FerroSelect […]


Cell and Gene Therapy, Industry

BioMarin’s application for haemophilia A gene therapy accepted by FDA

October 14, 2022

Via: PMLiVE

If approved, valoctocogene roxaparvovec would be the first gene therapy in the US for this indication. People living with haemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk of painful and potentially […]


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

Gene Therapy Helps Those With Rare Form of Blindness See Better at Night

October 13, 2022

Via: Drugs.com

An experimental gene therapy offers hope for rapid improvement in the night vision of adults who have a congenital form of childhood-onset blindness, researchers report. In this ongoing clinical trial at the Scheie Eye Institute in the Perelman School of […]


Cell and Gene Therapy, FDA, Industry, Regulations

FDA Expands Oversight of Cell and Gene Therapies

October 12, 2022

Via: Biopharm International

FDA’s Center for Biologics Evaluation and Research (CBER) is updating how it manages a growing volume of cellular and gene therapy development programs, seeking added resources and revisions in its oversight of these cutting-edge therapies. Most visible in the elevation […]


Mergers and Acquisitions

Solid Biosciences acquires AavantiBio, conducts $75 million private placement

October 4, 2022

Via: Pharmaphorum

AavantiBio, a precision genetic medicine company that was bankrolled by Solid’s rival Sarepta back in 2020, focuses on neuromuscular and cardiac rare diseases. Its key assets include product candidates for Duchenne muscular dystrophy (DMD), Friedreich’s ataxia, and BAG3 mediated dilated […]


Cell and Gene Therapy, Industry

BioMarin resubmits its hemophilia gene therapy to the FDA

September 30, 2022

Via: Biopharma Dive

Roctavian’s road back to the FDA has been long, as BioMarin has had to gather additional data in support of its therapy. Delays and new safety concerns further hampered the company’s resubmission plans. The treatment is for people with severe […]


Cell and Gene Therapy, Industry

Sarepta asks FDA to approve first gene therapy for Duchenne muscular dystrophy

September 29, 2022

Via: Biopharma Dive

Sarepta Therapeutics has asked the Food and Drug Administration to approve its gene therapy for Duchenne muscular dystrophy under accelerated review, the company said Thursday. The biotechnology company’s application for SRP-9001, also known as delandistrogene moxeparvovec, is primarily based on […]


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

Vertex, CRISPR prep filing for gene-editing blood disorder therapy

September 28, 2022

Via: Pharmaphorum

The rolling application for exagamglogene autotemcel (exa-cel) – formerly known as CTX001 – will start in November, while a filing in Europe is also expected by the end of the year, said the two partners. The time places exa-cel in […]


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

Pfizer, Sangamo resume haemophilia gene therapy trial

September 26, 2022

Via: Pharmaphorum

The phase 3 AFFINE trial was paused voluntarily by the two partners almost a year ago, and subsequently placed on hold by the FDA, after patients treated with the therapy produced higher than expected levels of Factor VIII – the […]


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

Pfizer, Sangamo set to resume gene therapy study after safety delay

September 23, 2022

Via: Biopharma Dive

Pfizer and Sangamo Therapeutics will soon resume a Phase 3 trial of their gene therapy for the blood disorder hemophilia A, nearly a year after safety concerns forced its suspension. Specifically, the companies said Thursday evening they plan to resume […]


Cell and Gene Therapy, Industry, Mergers and Acquisitions

GenScript and Avectas Partner on Next-Generation Cell Therapies

September 22, 2022

Via: GEN

GenScript and Avectas agreed to partner to develop an improved non-viral cell therapy manufacturing process. By combining Avectas’ cell engineering technology with GenScript’s expertise in synthetic long oligo production, the partnership aims to demonstrate a novel and efficient solution for […]


Cell and Gene Therapy, Industry, Mergers and Acquisitions

GenScript and Avectas link up to improve non-viral cell therapy processes

September 20, 2022

Via: Pharma Times

GenScript – the life-sciences research tools and services provider – and Avectas, a cell engineering technology company, are partnering to develop an improved non-viral cell therapy manufacturing process. The two companies are focusing on new methods for developing cell therapies […]


Cell and Gene Therapy, Industry

Back-to-back gene therapy approvals give Bluebird shot at survival

September 19, 2022

Via: Biopharma Dive

Until this August, just two gene therapies for inherited diseases were available in the U.S. Now, in the span of one month, that count has doubled, with the Food and Drug Administration approving new treatments for a rare blood condition […]


Cell and Gene Therapy, Industry

SparingVision raises €75m for eye disease gene therapies

September 14, 2022

Via: Pharmaphorum

The French biotech said the cash injection – which follows a €45 million Series A in 2020 – will also help it bring forward the first development candidate in a collaboration signed with gene-editing specialist Intellia Therapeutics last year, currently […]