KHK

Alnylam Highlights Significant Progress with Platform Innovation and Clinical Pipeline at R&D Day

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Wednesday, December 13, 2023
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, CNS, Hypertension, Cerebral amyloid angiopathy, Hepatocellular carcinoma, Gene, ALN, Cardiomyopathy, RNA interference, IND, Tissue, Liver, Sanofi, Amyloidosis, Gene silencing, Overweight, KHK, Patient, FGF21, CAA, Part, Obesity, T2DM, Neoplasm, Investigational New Drug, Therapy, LNP, Development, Shanda, Growth, HCC, Central nervous system, Type, TTR, Immunotherapy, Polyneuropathy, Haemophilia, Medication, Biology, Coagulopathy, Traffic barrier, Adipose tissue, Vaccine, Medical device, Pharmaceutical industry, Video game, Alnylam Pharmaceuticals, Capella, ATTR

We expect our track record of strong pipeline and commercial execution to continue through the end of 2025 and beyond,” said Akshay Vaishnaw, M.D., Ph.D., Chief Innovation Officer of Alnylam.

Key Points: 
  • We expect our track record of strong pipeline and commercial execution to continue through the end of 2025 and beyond,” said Akshay Vaishnaw, M.D., Ph.D., Chief Innovation Officer of Alnylam.
  • In addition, Alnylam will share an update on delivery advances driving ALN-BCAT, which will soon begin clinical testing in patients with hepatocellular carcinoma (HCC).
  • A peak mean TTR reduction of 97% was achieved at Day 29 and a mean TTR reduction of 93% was sustained at Day 180.
  • The Company’s R&D Day event will be held today, Wednesday, Dec. 13, from 8:30 a.m. to 12:45 p.m.

Alnylam Submits CTA Application for ALN-KHK, an Investigational RNAi Therapeutic for the Treatment of Type 2 Diabetes

Retrieved on: 
Wednesday, December 21, 2022
Science, Biotechnology, Research, Pharmaceutical, Health, Diabetes, Genetics, Clinical Trials, T2DM, Insulin resistance, RNA interference, Volunteering, KHK, Fructose, Health Canada, Overweight, ALN, Alnylam Pharmaceuticals, Medication, Patient, CTA, Type 2 diabetes, Fatty liver disease, Type 2, Therapy, Obesity, Diabetes, RNA, Pharmaceutical industry

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi (RNA interference) therapeutics company, announced today that the Company has submitted a Clinical Trial Authorization (CTA) application to Health Canada to initiate a Phase 1/2 study of ALN-KHK, an investigational RNAi therapeutic targeting ketohexokinase (KHK) for the treatment of Type 2 diabetes mellitus (T2DM).

Key Points: 
  • Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi (RNA interference) therapeutics company, announced today that the Company has submitted a Clinical Trial Authorization (CTA) application to Health Canada to initiate a Phase 1/2 study of ALN-KHK, an investigational RNAi therapeutic targeting ketohexokinase (KHK) for the treatment of Type 2 diabetes mellitus (T2DM).
  • “We are excited to advance ALN-KHK to the clinic as our first RNAi therapeutic to address the unmet medical need in Type 2 diabetes mellitus and potentially other metabolic disorders,” said Neil Wallace, Program Leader for the ALN-KHK program at Alnylam.
  • “Increased dietary fructose consumption, largely resulting from use of high-fructose corn syrup as a sweetener, is believed to contribute to the growing pandemic of obesity.
  • Fructose itself predisposes to fatty liver disease and thus, in the context of T2DM, aggravates insulin resistance.