The safety and efficacy of these products for the indication under study have not yet been established by the relevant regulatory authorities.
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Hepatorenal Syndrome Type 1 (HRS-1) is a life-threatening condition that consists of rapid deterioration in kidney function in individuals with cirrhosis or liver failure. HRS-1 affects from 10,000 to as many as 30,000 patients in the U.S. This condition can lead to multi-organ failure including acute kidney failure, and is often fatal unless a liver transplant is performed or the condition is reversed. There is no FDA-approved pharmacological therapy for treatment of HRS-1 in the U.S.
Reversal of HRS has been shown to increase survival and improve clinical outcomes. Terlipressin is the most widely studied and clinically accepted pharmacological therapy globally for patients with HRS-1.
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Graft versus Host Disease (GvHD) is a rare disorder that strikes bone marrow transplant or blood transfusion recipients whose immune system is deficient or suppressed. It can manifest as a skin rash, intestinal problems and liver dysfunction. It can be acute (sudden) or chronic (long lasting). Chronic GvHD lasts beyond 100 days and usually persists long after a bone marrow transplant.
Approximately 60% of bone marrow transplant patients develop GvHD, which is associated with high morbidity and mortality. Treatment with Extracorporeal Photopheresis for chronic GvHD is being studied in Japan.
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Partial thickness burns (also known as second degree burns) affect the top two layers of skin. These burns can change over time and either heal or worsen into a full thickness (or third degree burn) after initial treatment. Partial thickness burns are serious and can become infected or be complicated by non-healing or abnormal scarring. The current standard of care for deep burns (partial or full thickness) includes skin-grafting (or autografting), which is the surgical harvest of healthy skin from an uninjured site on the patient’s body followed by transplant to the wound. It results in a donor site wound that requires medical management of pain, and may result in infection and/or scarring.
Results from a previous study in deep partial thickness burns demonstrated that StrataGraft skin tissue closed wounds and reduced the need for autografting. StrataGraft skin tissue is being studied as an alternative to autografting.
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Niemann-Pick Type C (NPC) is a rare, neurodegenerative, and ultimately fatal disease that can present at any age. NPC is caused by mutations in either the NPC1 or NPC2 genes, resulting in the disruption of the trafficking of intracellular cholesterol, leading to intracellular lipid accumulation in various tissues, including the brain, liver, and spleen. Manifestations of the genetic disorder typically occur in childhood, with occasional late onset, and average diagnosis at ten years of age. NPC is usually fatal, and the majority of cases lead to death before age 20.
Results of a previous Adrabetadex study showed the potential for a disease-modifying effect based on slowing of progression on neurological, disease-specific outcomes measures and promising clinical improvements in patients with NPC. Adrabetadex is being studied further to assess safety and efficacy in patients with NPC.
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Hepatic encephalopathy is a critical neurocognitive complication of chronic liver disease. It is associated with elevated circulating ammonia levels stemming from end stage liver disease (cirrhosis) and is generally reversible. Symptoms range from mildly altered mental status to coma or death. Once a patient suffers an acute hepatic encephalopathy episode, prevention of recurrence is critical and may require chronic therapy.
A goal of hepatic encephalopathy treatment is to lower blood ammonia levels. MNK-6105 has a novel mechanism of action that may help in eliminating excess ammonia in the bloodstream by excretion through the kidneys. An intravenous form of MNK-6105 is being studied in hospitalized patients with acute hepatic encephalopathy.
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Full thickness burns (also known as third degree burns) affect all layers of the skin, and may cause damage down to underlying muscles. These burns carry severe risk for complications, such as infections, blood loss and shock, which could lead to organ failure or death. The current standard of care for severe burns is skin-grafting, or autografting, which is the surgical harvest of healthy skin from an uninjured site on the patient’s body followed by transplant to the wound. It results in a donor site wound that requires medical management of pain, and may result in infection and/or scarring.
A previous study in full thickness skin defects showed that StrataGraft skin tissue remained intact and viable throughout the seven-day placement period when it was used as a temporary cover to prepare the wound for skin-grafting. StrataGraft skin tissue is being studied further as a way to reduce the amount of autografting required in these patients.
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Hepatic encephalopathy is a critical neurocognitive complication of chronic liver disease. It is associated with elevated circulating ammonia levels stemming from end stage liver disease (cirrhosis) and is generally reversible. Symptoms range from mildly altered mental status to coma or death. Once a patient suffers an acute hepatic encephalopathy episode, prevention of recurrence is critical and may require chronic therapy.
A goal of hepatic encephalopathy treatment is to lower blood ammonia levels. MNK-6105 has a novel mechanism of action that may help in eliminating excess ammonia in the bloodstream by excretion through the kidneys. An intravenous form of MNK-6105 is being studied in hospitalized patients with acute hepatic encephalopathy.
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Diabetic foot ulcers (DFU) are open sores or wounds that occur on the bottom of the foot in approximately 15% of patients with diabetes and are the cause of more hospitalizations than any other complication of diabetes. Of those who develop a foot ulcer, approximately 6% will be hospitalized due to infection or other ulcer-related complication.
ExpressGraft (C9T1 skin tissue) is the first genetically engineered human skin substitute in development that expresses therapeutic proteins and may help reduce infection and accelerate wound healing. ExpressGraft (C9T1 skin tissue) has been shown in preclinical studies to augment one of the skin’s native antimicrobial defense mechanisms. It is being further evaluated for its potential to promote the healing of chronic wounds, including diabetic foot ulcers.
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ExpressGraft (pro-angiogenic VEGF) is the first genetically engineered human skin substitute in development that expresses therapeutic proteins and may help reduce infection and accelerate wound healing. The pro-angiogenic properties of ExpressGraft provide sustained expression of human vascular endothelial growth factors (VEGF) that help to promote neovascularization and tissue regeneration within complex skin defects while providing a barrier function.
Potential applications include chronic wounds such as venous leg and/or diabetic foot ulcers, as well as acute wounds such as traumatic wounds that may require stimulation for neovascularization.
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ExpressGraft (IL-12) is the first genetically engineered human skin substitute in development that expresses therapeutic proteins and may help reduce infection and accelerate wound healing based on preclinical research. The anti-tumor properties of ExpressGraft (anti-tumor IL-12) provide sustained expression of human IL-12 and may suppress the growth of human skin cancer and secrete wound healing factors that may stimulate tissue regeneration.
A potential application may be following a surgical procedure for removal of a localized skin cancer.
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The complement cascade is a group of proteins that are involved in the immune system and that play a role in the development of inflammation. These proteins are known to contribute to the pathogenesis of many diseases, including autoimmune diseases.
The RNAi drug target is designed to inhibit the complement cascade.
DFU: Diabetic Foot Ulcer, DPT: Deep Partial Thickness, FT: Full Thickness, GvHD: Graft vs Host Disease, HRS: Hepatorenal Syndrome, JP: Japan, RNAi: Ribonecleic Acid interference, VEGF: Vascular Endothelial Growth Factor
*NPXe Limited is developing xenon gas for inhalation for the treatment of Post-Cardiac Arrest Syndrome. Mallinckrodt has licensed the rights to market xenon gas for inhalation in North America, Australia and Japan upon approval in each country.
**Collaboration with Silence Therapeutics