Remove Gene Remove In-Vivo Remove Licensing Remove Trials
article thumbnail

The Significance of the MHRA Approval and Upcoming FDA Review of the First Gene Editing Treatment

Worldwide Clinical Trials

Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow. In June 2023, the U.S.

article thumbnail

10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

XTalks

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

Editas grabs orphan drug status for sickle cell disease CRISPR therapy

Pharmaceutical Technology

The US Food and Drug Administration (FDA) has granted an Orphan Drug Designation to Editas Medicine’s gene therapy EDIT-301 in sickle cell disease, based on an April 27 announcement. On January 19, Editas announced that it entered into a definitive agreement with Shoreline Biosciences for the licensing of its natural killer cell program.

article thumbnail

Bayer trumpets $1bn CRISPR deal with Mammoth Bio

pharmaphorum

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. CRISPR drugs can be used to modify the expression of disease-associated proteins in the body, for example, by correcting a mutation in a specific gene.

In-Vivo 52
article thumbnail

Biogen teams up with gene-editing startup Scribe on ALS programme

pharmaphorum

Scribe Therapeutics, a start-up focusing on gene-editing using CRISPR/Cas9, has burst onto the biotech scene with a $415 million deal with Biogen. It will also use a variation of CRISPR/Cas9 known as CRISPR/Casx which is smaller, making it easier to deliver using viral vectors for example, and more efficient at modifying genes in the body.

article thumbnail

The challenges and trends of cell & gene therapies 

Drug Discovery World

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. billion, compared to $19.9 Cancer is king .

article thumbnail

Birinapant licensing; AvantGen, IGM pairs up for anti-SARS-CoV-2 antibodies; BeiGene, Novartis to co-dvelop Tislelizumab; Valo raises USD190 M; Bluebird Bio Spins-off

Delveinsight

Medivir, IGM Biosciences enters into an exclusive licensing agreement for Birinapant. Medivir AB has entered into an exclusive licensing agreement with IGM Biosciences to receive global, exclusive development rights for Birinapant. million after Birinapant successfully becomes a part of the Phase I trials.