Liege, Belgium – EXO Biologics SA, a Belgian biotech company committed to developing biopharmaceuticals using exosomes to treat rare diseases with high unmet medical needs, today announces the worldwide launch of ExoXpertTM, a contract development and manufacturing organization (CDMO) specializing in exosomes. ExoXpert offers a MSC-based exosome manufacturing platform used in European clinical trials. ExoXpert is a wholly owned subsidiary of EXO BiologicsTM. [Read more…]
Deverra Therapeutics Granted FDA Regenerative Medicine Advanced Therapy and Orphan Drug Designations for DVX101 (Dilanubicel) for the Treatment of Acute Myeloid Leukemia
Designations Validate Preliminary Data and Enhances Ability to Offer Dilanubicel to Cancer Patients
SEATTLE, WA, March 19, 2024 – Deverra Therapeutics Inc., a clinical-stage biotechnology company developing allogeneic universal donor cell therapy treatments to fight cancer and other life-threatening diseases, today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT)designation to its lead candidate (dilanubicel) for use in first-line treatment for acute myeloid leukemia (AML). [Read more…]
What Is An RMAT? List of Publicly Announced RMAT Designations (89)
To date, what number of U.S. FDA-approved RMATs have been issued and to whom have they been awarded?
The answer is that 89 RMAT (Regenerative Medicine Advanced Therapy) designations have been publicly announced by biotech and pharma companies. However, the FDA states it has received 248 requests and issued 101, which means that a handful are not yet public knowledge. Therefore, a few companies are operating in stealth mode with regard to their RMAT designations and approximately 41% of RMAT applications get approved (101 approvals / 248 applications = 40.7%). [Read more…]
Asgard Therapeutics Snags €30M to Develop In Vivo Direct Cell Reprogramming Technologies for Cancer
€30M Series A financing will advance a novel, off-the-shelf gene therapy (AT-108) that directly reprograms tumor cells into antigen-presenting dendritic cells.
Lead program is AT-108, a first-in-class, off-the-shelf intra-tumoral therapy that leads to personalized and potent anti-cancer immune responses.
Co-led by RV Invest and Johnson & Johnson Innovation – JJDC, Inc. with participation from existing investors Novo Holdings, Boehringer Ingelheim Venture Fund and Industrifonden.
Direct Cell Reprogramming: What Is It and How Does It Work?
Direct cell reprogramming is the in vitro (laboratory) or in vivo (in the body) reprogramming of somatic cells into other cell types without the need for an intermediate pluripotent state. Another term for direct cell reprogramming is transdifferentiation, which is the direct conversion of one differentiated cell type into another.
There has been tremendous activity over the last decade in the development of stem cells therapeutics, which have relied on stem cell differentiation protocols, as well as techniques for direct cell reprogramming. Today, there are over 7,149+ stem cell trials registered on ClinicalTrials.gov and stem cell publications have increased dramatically in recent years, recently surpassing 500,000+ on PubMed.gov.
However, for all their promise, stem cells are yet to deliver on this therapeutic potential, and this failure appears due to various technical challenges that still need to be overcome. [Read more…]