XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. Both therapies will be available in early 2024.

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pharmaphorum

AUGUST 24, 2021

Vertex Pharma has ramped up its involvement in gene-editing medicines for the third time in a matter of months, agreeing a partnership with CRISPR specialist Arbor Biotechnologies that could be worth up to $1.2 If approved, it could mount a challenge to bluebird bio’s gene therapy Zynteglo, which is already approved in Europe.

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pharmaphorum

SEPTEMBER 14, 2022

SparingVision has raised €75 million in second-round funding that will be used to fund clinical trials of gene therapies for ocular diseases retinitis pigmentosa (RP) and dry age-related macular degeneration (AMD). The post SparingVision raises €75m for eye disease gene therapies appeared first on.

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Scienmag

APRIL 21, 2021

New approach to gene therapy can correct any disease-causing mutation within a gene UNIVERSITY PARK, Pa.

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pharmaphorum

JANUARY 10, 2022

Bayer has bolstered its cell and gene therapy platform by securing access to a CRISPR-based gene-editing platform developed by US biotech Mammoth Biosciences. The post Bayer trumpets $1bn CRISPR deal with Mammoth Bio appeared first on.

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pharmaphorum

DECEMBER 6, 2020

A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has achieved “remarkable” improvements in patients with beta thalassaemia and sickle cell disease in an early-stage trial reported at the American Society of Haematology (ASH) annual meeting.

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Gene Editing Gene In-Vivo Genetics 52

pharmaphorum

FEBRUARY 17, 2022

The drug – which costs $125,000 at US list prices – can be used either alone or in combination with hydroxyurea, a well-established therapy for the disease. CRISPR Therapeutics and Vertex are also in the running with their gene-editing candidate CTX001, in phase 1/2 trials which are due to generate final results later this year.

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Drugs Gene Therapy Gene Editing Gene 59

XTalks

DECEMBER 29, 2022

For example, on the pharma and biotech side, there has been growing demand for new drugs and biologics, from new classes of dual-action diabetes and weight loss drugs like Ozempic and Mounjaro to life-saving gene therapies. 1. Cell and Gene Therapies. Here are four life sciences trends to look out for in 2023.

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The Pharma Data

DECEMBER 13, 2020

“Our company and R&D portfolio are entering into an exciting phase, as evidenced by the recent close of Series B financing and submission of the first gene editing product IND in China,” said Dong Wei, Ph.D.?CEO Prior to joining EdiGene, he was Vice President of KLUS Pharma and focused on cell therapy and new technologies.

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XTalks

MAY 4, 2021

The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies.

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DNA Gene Gene Silencing Genome 92

XTalks

MAY 4, 2021

The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies.

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