Merck Signs MoU with Gene Therapy Research Institution to Advance Gene Therapy Production
Merck, a prominent science and technology company, has recently signed a non-binding Memorandum of Understanding (MoU) with Gene Therapy Research Institution Co., Ltd. (GTRI), a Japanese clinical-stage biotech start-up focused on developing gene therapies using adeno-associated virus (AAV) vectors. This collaboration marks one of the first clinical-stage applications of Merck’s Sf-RVN® Insect Cell Line for […]
Alkaline Phosphatase: Potential cure for acute kidney injury?
Acute kidney injury (AKI) affects 20-50% of all hospitalized patients. It is characterized by an increase in serum creatinine and decreased urine output. AKI has mortality rate of >50%. Many factors are responsible for AKI like trauma, cardiovascular surgery, administration of nephrotoxic drugs, but sepsis associated AKI is the prominent one. Current treatment plans include […]
Mito!! Give me the power to rejuvenate!
We all know that mitochondria are the powerhouse of cells because they generate energy from food. However, we did not know their involvement in cellular regeneration. Scientists at UT Southwestern Medical Center determined that liver cells with damaged mitochondria have less ability to regenerate when injured. Henceforth, allowing cells with undamaged mitochondria to grow more, […]
J For Juvenile CLN3 Disease
Juvenile CLN3 disease, commonly known as Juvenile Batten Disease, is an extremely rare and fatal inherited disorder primarily affecting the nervous system in children. The disease is caused by mutations in the CLN3 gene, which encodes the CLN3 protein. More than sixty-seven different mutations in the CLN3 gene have been identified as responsible for Juvenile […]
Forget Me Not: New Alzheimer’s Drug from Eli Lilly Gets FDA Nod
The FDA approved donanemab, a new drug for early-stage Alzheimer’s disease. I remember my grandmother, her summer house filled with the smell of freshly baked cookies, the air vibrating with the laughter of my cousins rolling on the floor from her silly jokes. It was the best time of our childhood. I wish it remained […]
FDA Grants Accelerated Approval to Epcoritamab-bysp for Relapsed or Refractory Follicular Lymphoma
The U.S. Food and Drug Administration (FDA) has granted accelerated approval to epcoritamab-bysp (Epkinly), developed by Genmab, for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy. This approval marks a significant advancement in the treatment options for FL, a type of non-Hodgkin lymphoma. […]
I for I-Cell Disease- Part II
I-cell disease or Mucolipidosis Type II (MLII) is a devastating rare genetic disorder characterized by severe multisystemic involvement and a limited lifespan. It is a severe lysosomal storage disorder caused by mutations in the GNPTAB gene located on chromosome 12q23.2. This gene encodes the alpha and beta subunits of N-acetylglucosamine-1-phosphotransferase (GlcNAc-1-phosphotransferase), which plays a crucial […]
FDA Grants Accelerated Approval to Adagrasib with Cetuximab for KRAS G12C-Mutated Colorectal Cancer
The Food and Drug Administration (FDA) has granted accelerated approval for the combination of adagrasib (Krazati; Mirati Therapeutics, Inc.) and cetuximab for the treatment of adults with KRAS G12C-mutated locally advanced or metastatic colorectal cancer (CRC). This approval applies to patients who have previously been treated with chemotherapy regimens based on fluoropyrimidine, oxaliplatin, and irinotecan, […]
I for I-Cell Disease- Part I
I-cell disease, also known as Mucolipidosis type II, is a severe rare lysosomal storage disorder caused by mutations in the GNPTAB gene located on chromosome 12q23.2. This gene encodes the alpha and beta subunits of N-acetylglucosamine-1-phosphotransferase, which plays a critical role in processing and packaging lysosomal enzymes by tagging them with mannose-6-phosphate in the Golgi […]
Iqirvo Clears FDA Hurdle, Offering New Relief for Primary Biliary Cholangitis Patients
The FDA approves Iqirvo (elafibranor) for primary biliary cholangitis (PBC) treatment, offering a new option for patients intolerant to UDCA. What is Primary Biliary Cholangitis (PBC)? A case study from December 2023 reports a 19-year-old male with multiple congenital abnormalities and an intellectual disability suffered from pruritus for 5 years. Tests revealed elevated levels of […]
H for Huntington’s Disease- Part II
Huntington’s disease (HD) is a debilitating neurodegenerative condition caused by an abnormal expansion of CAG triplets in the huntingtin (HTT) gene, leading to the progressive deterioration of GABAergic neurons in the brain’s central regions. As of now, there is no cure for HD, and treatment focuses on symptom management and improving quality of life. The […]
FDA Approves Blinatumomab as Consolidation Therapy for CD19-Positive Philadelphia Chromosome-Negative B-Cell Precursor Acute Lymphoblastic Leukemia
The Food and Drug Administration (FDA) has granted approval for blinatumomab (Blincyto, Amgen) for use in adult and pediatric patients aged one month and older with CD19-positive Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia (Ph-negative BCP ALL) during the consolidation phase of multiphase chemotherapy. This decision marks a significant advancement in the treatment regimen for […]
H for Huntington’s Disease – Part I
Huntington’s disease (HD) is a rare, inherited neurodegenerative disorder affecting the central nervous system. Characterized by involuntary movements, behavioral disturbances, and dementia, HD typically manifests between ages 30 and 50, though juvenile cases also occur. Symptoms include chorea, dystonia, cognitive decline, psychiatric issues, and difficulties with speech and swallowing. The disease leads to complete dependency […]
FDA Grants Accelerated Approval to Repotrectinib for NTRK Fusion-Positive Tumors
The Food and Drug Administration (FDA) has granted accelerated approval to repotrectinib (AUGTYRO, Bristol-Myers Squibb; BMS) for the treatment of adult and pediatric patients aged 12 and older with solid tumors harboring a neurotrophic tyrosine receptor kinase (NTRK) gene fusion. This approval covers patients with locally advanced or metastatic tumors for whom surgical resection would […]
Breaking Barriers in Rare Disease Treatment: FDA Approves Bkemv as First Interchangeable Biosimilar
The US Food and Drug Administration (FDA) has approved Amgen’s Bkemv as the first interchangeable biosimilar to Soliris for treating rare diseases, such as paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). The FDA has approved Bkemv, a biosimilar, as the first interchangeable treatment for two rare and debilitating diseases: paroxysmal nocturnal hemoglobinuria […]
US FDA Approves Breyanzi for Relapsed or Refractory Mantle Cell Lymphoma
The U.S. Food and Drug Administration (FDA) has approved lisocabtagene maraleucel (Breyanzi), developed by Juno Therapeutics, Inc., for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL) who have undergone at least two prior lines of systemic therapy, including a Bruton tyrosine kinase inhibitor (BTKi). This approval marks a significant advancement […]
G for Gaucher Disease – Part II
Gaucher disease results from mutations in the GBA gene, disrupting the breakdown of glucocerebroside and causing its accumulation, leading to cellular damage. Efforts to repair this faulty “switch” have long been pursued in medical research. This disease though currently incurable, can be managed effectively with available therapies. Type 1 Gaucher patients can lead full lives, […]
FDA Greenlights mRESVIA: Moderna’s Breakthrough in RSV Prevention
Moderna’s mRESVIA, an mRNA vaccine, has received FDA approval, a significant milestone in the fight against respiratory syncytial virus (RSV). June 9th, 2024: In a significant development for public health, Moderna has received approval from the U.S. Food and Drug Administration (FDA) for its new respiratory syncytial virus (RSV) vaccine, mRESVIA. This marks a major […]
Eyeing a Brighter Future: Two New Biosimilars Approved for Macular Edema
FDA approves biosimilars Yesafili and Opuviz for Diabetic Macular Edema, offering affordable treatment options. Breakthrough in Diabetic Macular Edema Treatment May 25th, 2024: The FDA’s recent approval of two new biosimilars, Yesafili (aflibercept-jbvf) and Opuviz (aflibercept-yszy), marks a significant breakthrough in the treatment of Diabetic Retinopathy. These biosimilars are interchangeable with Eylea (Aflibercept), a biologic […]
G for Gaucher Disease – Part I
Gaucher disease is a rare, genetic metabolic disorder where glucocerebrosidase deficiency lead to accumulation of fat-laden “Gaucher cells” in areas like the spleen, liver and bone marrow. Gaucher cells are normal macrophages which become full of unprocessed glucocerebroside and accumulate in the organs causing dysfunction and inflammation. This is the second most severe type of […]
Follicular Lymphoma Treatment Option Gains FDA Approval: Lisocabtagene Maraleucel
The Food and Drug Administration (FDA) granted accelerated approval to lisocabtagene maraleucel (Breyanzi, Juno Therapeutics). Lisocabtagene maraleucel is a chimeric antigen receptor (CAR) T-cell therapy. CAR T-cell therapies are a type of immunotherapy that involves engineering a patient’s T cells to recognize and attack cancer cells. Lisocabtagene maraleucel T cells are specifically designed to target […]
Insulin Efsitora Alfa-Substitute for Continual Insulin Administration
Eli Lilly Efistora has showed positive outcomes from QWINT-2 and QWINT-4 phase 3 clinical trials. The trials discerned impact of the once weekly insulin efsitora in patients with T2 diabetes using insulin for the first time. The trials compared results with patients requiring multiple daily insulin injections. Although results of efsitora consumption are not superior […]
F for Fanconi Anemia – Part II
Fanconi anemia (FA) is a rare genetic disorder characterized by bone marrow failure, birth defects, and a heightened risk of cancer. Symptoms typically emerge around age 7, though diagnosis may occur later when cytopenia is detected. The disorder’s average lifespan is between 20 and 30 years, with most patients developing bone marrow failure (BMF) by […]
F for Fanconi Anemia – Part I
Fanconi Anemia (FA) is a rare autosomal recessive disorder caused due to mutations in any of the known 23 genes that play a role in the DNA repair pathway. Defect in the DNA repair in all cells of the body results in accumulation of errors in these cells over time, thus making people living with […]
FDA Grants Accelerated Approval to Tarlatamab-dlle for ES-SCLC
The Food and Drug Administration (FDA) has granted accelerated approval to tarlatamab-dlle (Imdelltra, Amgen, Inc.) for the treatment of extensive stage small cell lung cancer (ES-SCLC) in patients with disease progression following platinum-based chemotherapy. Tarlatamab-dlle is an innovative bi-specific T-cell engager designed to direct the body’s immune response specifically towards cancer cells. This mechanism allows […]
E for Erdheim-Chester Disease- Part II
Diagnosing Erdheim-Chester Disease (ECD), presents significant challenges due to its rarity and varied clinical presentations. Successful diagnosis typically involves integrating descriptive pathology, clinical observations, and radiographic findings. Patients often undergo multiple biopsies, leading to delays in diagnosis and treatment initiation, with the average time from symptom onset to diagnosis ranging from months to years. Determining […]
Sanofi & Novavax Partner on COVID-19 and Flu Combination Vaccines
Sanofi and Novavax, two leading companies in the field of vaccine development, have recently announced a significant co-exclusive licensing agreement aimed at broadening access to COVID-19 vaccines and exploring novel combination vaccines targeting both COVID-19 and influenza. This collaboration underscores the companies’ commitment to advancing public health by leveraging their respective strengths in vaccine research, […]
Lixisenatide has added benefits for treating Parkinson disease
New Diabetic Drug Lixisenatide has found its new purpose to treat Parkinson disease. The studies published in New England Journal of Medicine suggested that lixisentide therapy attenuated the progression of motor disability in patients diagnosed with Parkinson disease in less than 3 years (Meissner et al., 2024). These benefits were associated with gastrointestinal side effects. […]
E for Erdheim-Chester Disease Part-1
Erdheim-Chester Disease (ECD) is an extremely rare multisystem adult-onset histiocytic disorder which is a type of a slow-growing blood cancer. Histiocytes are large phagocytic cells, typically tasked with combatting infections. In this disease, there is an excessive accumulation of histiocytes which infiltrate the body’s loose connective tissue, triggering inflammation and thickening of the affected areas. […]
Accelerated Approval of Tovorafenib for Pediatric Low-Grade Glioma: A Breakthrough in BRAF-Altered Therapy
Pediatric low-grade glioma (LGG) with BRAF alterations presents a significant therapeutic challenge, lacking approved systemic treatments. However, a recent milestone has been achieved with the Food and Drug Administration’s (FDA) accelerated approval of tovorafenib (Ojemda, Day One Biopharmaceuticals, Inc.) for patients aged 6 months and older with relapsed or refractory pediatric LGG harboring BRAF fusions, […]