Top Five Oligonucleotide Therapeutics Collaborations of 2024: Driving Innovation in RNA-Targeted Drug Development

• Time:January 13, 2024
  
• Content:Ribo Life Science Collaborates with Boehringer Ingelheim to Develop Innovative Small Nucleic Acid Therapies for NASH/MASH Using Ribo-GalStartm Technology
• Company Profile:Ribo Life Science is a leading company in China for the research and development of small nucleic acid drugs, focusing on the R&D, production, and commercialization of such drugs. It has strong technical capabilities in R&D, with multiple self-developed technology platforms. Its R&D pipeline is diverse, covering various disease areas.

Ribo Life Science R&D Pipeline

• Time:January 3, 2024
  
• Content:Argo Biopharma Partners with Novartis to Develop Innovative Small Nucleic Acid Therapies for Cardiovascular Diseases Using Argo's Proprietary RNAi Technology Platform. Argo will receive an upfront payment of $185 million from Novartis and is eligible for potential option fees, milestone payments, and tiered royalties on commercial sales. The total potential value of the deal is up to $4.165 billion. Under the collaboration agreement, Argo has exclusively licensed the global development and commercialization rights of a Phase I clinical product targeting cardiovascular diseases to Novartis. Additionally, Novartis will have the option to license compounds targeting up to two additional cardiovascular disease-related targets. Meanwhile, Argo has also exclusively licensed the development and commercialization rights outside of Greater China for a Phase I/IIa clinical-stage project for cardiovascular disease treatment to Novartis.
• Company Profile:Argo Biopharma focuses on the research and development of innovative drugs, achieving significant results in the technological innovation and clinical translation of small nucleic acid drugs, particularly excelling in the development of cardiovascular disease treatments. Its R&D pipeline is concentrated on cardiovascular diseases, with multiple small nucleic acid drugs at various stages of development. A small nucleic acid drug targeting coronary heart disease has entered the late stage of preclinical research, with the potential to reduce vascular plaque formation by inhibiting specific gene expression. Another drug for arrhythmia is in the early exploratory stage, aiming to correct cardiac electrophysiological abnormalities at the genetic level.

Argo's Partial R&D Pipeline

• Time:June 3
• Content:QurAlis Corporation Announces Exclusive Licensing Agreement with Eli Lilly for Global Rights to Develop and Commercialize QRL-204, a Potential Best-in-Class Splicing Modulator Antisense Oligonucleotide Designed to Restore UNC13A Function in ALS, FTD, and Other Neurodegenerative Diseases. QurAlis will receive a $45 million upfront payment, additional equity investment, and is eligible for up to $577 million in future milestone payments and tiered royalties on net sales.
• Company Profile:QurAlis is an American clinical-stage biotechnology company founded in 2016, dedicated to developing breakthrough precision drugs for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases with genetically validated targets. By leveraging its proprietary platform and biomarkers, the company designs and develops drugs that directly target disease-causing genetic alterations.

  
  

R&D Pipeline

• Time:November 26, 2024
  
• Content:Arrowhead Pharmaceuticals Reaches Global Licensing and Collaboration Agreement with Sarepta Therapeutics. The agreement covers multiple clinical and preclinical programs targeting rare genetic diseases of the muscle, central nervous system (CNS), and lungs, with up to 13 programs included. Arrowhead will receive $825 million, including $500 million in cash and $325 million in equity investment, as well as nearly $300 million in short-term milestone payments related to current clinical programs. Over the next five years, Sarepta will also pay an additional $250 million through annual payments of $50 million. Furthermore, the agreement includes approximately $10 billion in future milestone payments.
• Company Profile:Arrowhead Pharmaceuticals has deep technical expertise in the field of small nucleic acid drug development, with a particular focus on creating innovative treatments for rare diseases. Its R&D pipeline centers around rare diseases, targeting various rare genetic disorders affecting muscles, the central nervous system, and the lungs. The company’s small nucleic acid drug for Duchenne muscular dystrophy is in a critical phase of clinical trials, aiming to alleviate muscle atrophy symptoms by improving gene expression.

Arrowhead Partial R&D Pipeline

• Time:December 30
  
• Content:Huadong Medicine and Syncon Pharmaceuticals Announce Strategic Collaboration to Co-develop SNK-2726, a Small Nucleic Acid Candidate Targeting Angiotensinogen, for the Treatment of Hypertension. Huadong Medicine will obtain an exclusive option for the development, registration, manufacturing, and commercialization of SNK-2726 in Greater China.
• Company Profile:SNK Pharmaceuticals was founded in 2021, focusing on the research and development of novel small nucleic acid drugs and clinical studies. It is committed to developing RNA-targeted therapies and possesses innovative delivery technologies targeting the liver and extrahepatic organs. Its R&D pipeline covers areas such as cardiovascular diseases, metabolic diseases, rare diseases, and central nervous system disorders. The company has several product pipelines with first-in-class or best-in-class potential, including SNK-2726, an siRNA candidate drug targeting angiotensinogen for the treatment of hypertension, and SNK396, China's first siRNA candidate drug targeting a specific site, which has completed dosing of its first enrolled subject.

  
  

R&D Pipeline

Reference: Public reports