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Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA geneediting therapies. The company will also receive tiered royalties on the worldwide net sales of products developed from the collaboration.
Asklepios BioPharmaceutical has entered a research partnership and option agreement with ReCode Therapeutics for exploring its single-vector gene-editing platform. The new solution will enable complete gene insertion by delivering the gene-editing tool and DNA as mixed cargo to desired targets in one LNP.
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. billion product if it gets approved for both indications.
The potential to develop treatments with CRISPR geneediting is hinged on its ability to add, delete or replace elements within a target DNA sequence. Cancer is a geneticdisease, caused by certain changes in the way that genes control cell function, such as how they grow and divide. Why cancer? The pipeline.
“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 geneeditingproduct IND in China,” said Dong Wei, Ph.D.?CEO Dr. Zhang completed his postdoctoral fellowship at Harvard Medical School/Boston Children’s Hospital.
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 in the early-discovery stage.
Gene therapy is a new therapeutic approach in which genes are used to treat or prevent diseases. It is a comprehensive term which encompasses a large variety of therapy products including viral and bacterial vectors, plasmid DNA, human geneediting technology, and patient-specific cellular gene therapy.
While Lyfgenia delivers a functional copy of hemoglobin A to replace the abnormal one in hematopoietic (blood) stem cells via a lentiviral vector, Casgevy edits the HBB gene using CRISPR technology to increase the production of fetal hemoglobin in blood stem cells. Both therapies will be available in early 2024.
In 2022 alone, the US regulator approved four new gene therapies, showing the high interest in getting these therapies to market. In 2023, a number of gene therapies are expected to get the FDA green light. Given their precise targeting, gene therapies have largely been focused on oncology indications followed by rare geneticdiseases.
After the success of its first commercial vaccine product, Moderna has developed an mRNA-based vaccine against influenza, for which it recently released early positive safety and efficacy data. The gene-editing tool CRISPR edits DNA using specialized strands of guide RNA and endogenous cellular mechanisms of RNA interference.
The company has also stated, at the same time as the study was announced, that it will look at developing mRNA technology in oncology and geneticdiseases. This not only allowed Pfizer to establish a COVID-19 vaccine but the company also recently announced that it had begun a study testing an mRNA flu vaccine.
Medical device manufacturers are leveraging these technologies to enhance their products, supporting healthcare providers and improving patient outcomes. RNA-based therapies hold the potential to offer new treatment options for diseases with limited or inadequate therapeutic alternatives.
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