Orphan Medicine Weekly | Amjin, Novarma, Asathe Pharmaceuticals and other companies 9 therapies shortlisted

Drugs: AVXS-401 Research and Development Enterprise: Novartis Gene Therapies Treatment Of Disease: Friedreich's Ataxia, FA Introduction: FA is caused by repeated amplification of triple nucleotides in the FXN gene encoded frataxin, which can lead to a significant reduction in frataxin protein levels.
lack of frataxin can cause iron to accumulate in tissues, affecting cells with high energy needs in tissues such as the heart and brain.
AVXS-401 is a gene therapy developed by Novortis Gene Therapies that uses AAV9 adenovirus (AAV) vectors to deliver functional copies of the FXN gene to restore the production of frataxin in highly affected tissues.
early toxicity studies in healthy mice have shown that the AVXS-401 has good safety and tolerance.
efficacy studies have shown that in FA mouse models lacking frataxin in the central nervous system, a single in-brain (ICV) injection of low-dose AVXS-401 improved behavior and promoted neuroglial cell growth.
in mouse models with a lack of frataxin in the heart, the AVXS-401 restores heart function, extends the mid-life by more than three times, and prevents the onset of cardiovascular disease.
When the therapeutic dose is increased proportionally and given in non-human primates, AVXS-401 achieves long-lasting expression of frataxin in the central nervous system and heart, and the effect is still evident after 6 months of treatment.
Drugs: Carfilzomib Research and Development: Amgen Treatment: Acute Lymphocytic Leukemia (ALL) Introduction: Carfilzomib has been approved by the FDA to treat relapsed or refractic multiple myeloma with other treatments.
It is a protease inhibitor, myeloma cells rely on proteases to degrade excess proteins in order to reuse the amino acids produced by degradation to continue to grow and reproduce, while kafizomi causes many proteins to accumulate in cells by blocking the proteases of cells, eventually leading to myeloma cell death.
Drugs: ATI-450 Research and Development Enterprise: Aclaris Therapeutics Treatment of Diseases: Cryopyrin-associated periodic syndrome (Cryopyrin-associated periodic syndrome, CAPS) Introduction: ATI-450 is an oral small molecule inhibitor targeting the p38 alpha fissure active protein kinase active protein kinase 2 (MK2) inflammatory signaling path.
path is used to drive the expression of inflammatory signals such as cytokines, coercion factors, and substring metal proteases.
key inflammatory cytokines driven by this pathline include tumor necrotic factor α (TNF alpha), lecytokine-1 alpha and -1 beta (IL1 alpha and IL1 beta) and lelutin-mediate-6 (IL6).
based on this mechanism, Aclaris is developing ATI-450 as a potential treatment for immune inflammatory diseases.
Drugs: APG-2575 Research and Development Enterprise: Assason Pharmaceuticals Treatment of Diseases: Follicular lymphoma (FL) Introduction: Follicular lymphoma is the second most common type of non-Hodgkin's lymphoma (NHL) and is an inert lymphoma, accounting for about 22% of NHL cases.
although there are treatments to improve total survival, FL is considered an incurable malignant tumor, showing patterns of disease progression that are constantly recurring and remission.
APG-2575 is a new oral Bcl-2 selective small molecule inhibitor developed by Asaan Pharmaceuticals to restore the tumor cell programmed death mechanism (apoptosis) by selectively inhibiting Bcl-2 protein, thereby killing tumors and intended to be used to treat a variety of blood malignancies.
previously, it had been eligible for four orphan drugs for the treatment of Fahrenheit globulinemia (WM), chronic lymphoblastic leukemia (CLL), multiple myeloma (MM) and acute myeloid leukemia (AML).
same time, APG-2575 has been licensed for phase 1b/2 clinical trials in the United States, China and Australia, and is advancing the clinical development of multiple blood tumor adaptations worldwide simultaneously.
Drugs: Uttroside B Research and Development Enterprise: Q BioMed Treatment of Diseases: Hepatocellular Carcinoma (HCC) Introduction: Uttroside B is a saponin derived from black-chain mold that is a natural small molecule compound that affects the activity of JNK's phosphate and caspase, thereby regulating signaling path paths associated with cell survival and apoptosis.
Uttroside B produces cytotoxicity for a variety of liver cancer cell types and is well treated in preclinical studies.
Drugs: T Lymphocytes from CD34-plus hematopoietic stem cells Research and development enterprise: Smart Immune Treatment Of Diseases: Enhanced Cell Implantation for Patients Receiving Hematopoietic Stem Cell TransplantAtion Introduction: Smart Immune's proprietary technology is based on a bionic introphy thymus platform that differentiates CD34-plus stem cells from cord blood or epithelial hematopoietic stem cells into T lymphocytes.
when these T lymphocytes are entered into the body, they migrate to the patient's thymus, where they quickly mature and gain the ability to kill viruses and cancer cells without attacking healthy cells.
this can greatly reduce the risk of graft resistance to host disease.
this cell therapy has a synergetic effect on the thymus epithroid, expressing cytokines, allowing the thymus epithal to recombine and regenerate, thereby significantly speeding up cell implantation.
Drugs: AVR-48 Research Research Treatment of Diseases: Prevention of Bronchopulmonary Dysplasia, BPD Introduction: triculmonary dysplasia is a chronic lung disease that occurs mostly in premature infants and patients often require oxygen therapy.
AyuVis is developing a new small molecule immunomodulant, AVR-48, that enhances the body's ability to fight inflammation and restore lung function through intravenous administering, helping to reduce or prevent BPD in premature children and reducing hospital stays.
anti-inflammatory properties of AVR-48 are different from current steroid therapies, which do not inhibit and synergetic normal immune function.
: This article is intended to introduce medical and health research, not treatment options recommended.
if you need guidance on treatment options, visit a regular hospital.
resources: novartis Gene Therapies. Retrieved Jan 22, 2021,from [2] Amgen. Retrieved Jan 22, 2021,from [3] Aclaris. Retrieved Jan 22, 2021, from the 9th FDA Orphan Drug Qualification, the largest in China. Retrieved Jan 22, 2021,from [5] Q BioMed. Retrieved Jan 22, 2021,from [6] Smart Immune. Retrieved Jan 22, 2021,from [7] Ayuvis. Retrieved Jan 22, 2021,from