11 therapies including oligonucleotide drugs and "ready-to-use" T lymphocyte therapy are shortlisted

Last week, the FDA issued 11 orphan drug qualifications, including 6 small molecule targeted drugs, 1 antibody-conjugated drug, 1 stem cell therapy, 1 gene therapy, 1 oligonucleotide, and 1 immune cell therapy.

Drug: CLBS12

Research and development company: Caladrius Biosciences

Treatment of diseases: Thromboangiitis obliterans

Introduction: CD34 is a cell surface protein expressed by specific monocytes in the bone marrow and blood circulation.

Drug: Cavrotolimod (AST-008)

R&D company: Exicure

Treatment of diseases: Merkel cell carcinoma (Merkel cell carcinoma, MCC)

Introduction: Merkel Cell Carcinoma (MCC), also known as cutaneous neuroendocrine carcinoma, is a rare and highly aggressive tumor that occurs at the site of sun damage.

Drug: Tesomet

R&D company: Saniona

Treatment of diseases: Prader-Willi syndrome (Prader-Willi syndrome, PWS)

Introduction: Prader-Willi syndrome (PWS) is a rare genetic disease, mainly due to the loss of a specific segment on chromosome 15, which causes the hypothalamic area of ​​the brain that manages appetite/satisfaction to malfunction.

Drug: BA3011

Research and development company: BioAlta

Treatment of diseases: soft tissue sarcoma (STS)

Introduction: Soft tissue sarcoma (STS) is a group of malignant tumors originating from connective tissues such as mucus, fibers, fat, smooth muscle, synovium, striated muscle, mesothelium, blood vessels and lymphatic vessels, including nerve tissue tumors originating from the neuroectoderm.

Drug: GDX012

Research and development company: GammaDelta Therapeutics

Treatment of diseases: Acute myeloid leukemia (AML)

Introduction: GDX012 is an allogeneic therapy based on γδ T lymphocytes.

Note: This article is intended to introduce medical and health research, not a treatment plan recommendation.

Reference materials:

[1] Kangfang Bio.

[3] AdAlta.

[4] Linnaeus Therapeutics.