Hongene Biotech Corporation

We are pleased to announce that Hongene has established a strategic collaboration with ReciBioPharm, a leading advanced therapeutics CDMO services provider, to enhance gene editing drug manufacturing capabilities!   The strategic alliance creates a unique solution for innovators in the gene editing field. ReciBioPharm will expand its service portfolio to include sgRNA manufacturing, utilizing Hongene's innovative synthesis process, providing plasmid, mRNA, sgRNA, LNP, and fill-finish services in a single facility. This will streamline the entire development and manufacturing process, mitigating the logistical complexities of dealing with multiple CDMOs, and ultimately accelerating the path for drug development.   Vikas Gupta, President of ReciBioPharm, stated: "Our collaboration with Hongene Biotech marks an important step forward for ReciBioPharm, enabling us to offer comprehensive, seamless solutions in the rapidly evolving gene editing sector. The combination of Hongene’s high-quality raw materials and synthesis methods with our capabilities will provide our clients with an unmatched portfolio of services in this developing field."   Echoing the sentiment, Wei Jiang, CEO at Hongene Biotech, shared: "Our partnership with ReciBioPharm is set to redefine the manufacturing space for gene editing. Our cutting-edge sgRNA synthesis technology, combined with ReciBioPharm's expertise in end-to-end mRNA and drug product manufacture, promises to bring a unique solution that has significant value to the healthcare field."   Find out more detailed information: https://lnkd.in/erikB-nR   #Breakingnews #GeneEditing #GeneTherapy #sgRNA #RNA

A map of global public perspectives on mRNA vaccines and therapeutics reflects the challenges associated with the novelty and ongoing misinformation about mRNA technology. The development and rollout of mRNA vaccines during the COVID-19 pandemic marked a significant advancement in vaccinology. Yet, public hesitancy towards vaccination was prevalent. Researchers from the Laboratory of Data Discovery for Health Limited (D²4H) and the London School of Hygiene and Tropical Medicine conducted a social listening analysis to assess attitudes towards mRNA vaccines and therapeutics on Twitter from June 2022 to May 2023. Results revealed widespread negative sentiment and a global lack of confidence in the safety, effectiveness, and trustworthiness of mRNA vaccines and therapeutics, with frequent discussions of severe vaccine side effects, rumors, and misinformation. Concerns regarding various side effects after vaccination and the subsequent spread of misinformation about mRNA vaccines contribute to a decline in confidence over time. This highlights the importance of public awareness and education on mRNA technology. It is essential to clearly explain how mRNA-based therapies and vaccines function and to effectively address public concerns with a joint effort to ensure that public discourse is informed by correct and verified information. #mRNAvaccine #mRNAtherapeutics #RNAtherapeutics #mRNA #vaccines Image credit to the original article: https://lnkd.in/e6Rxs2GT

Chemoenzymatic ligation is a promising approach for oligonucleotide and siRNA API manufacture, offering enhanced purity, increased yield, and reduced environmental impact. This innovative technology provides an optimal solution to meet the anticipated surge in demand for siRNA drugs over the coming decade. 🧬𝗪𝗲 𝘂𝘀𝗲 𝘁𝘄𝗼 𝗿𝗼𝗯𝘂𝘀𝘁 𝗺𝗲𝘁𝗵𝗼𝗱𝘀 𝗼𝗳 𝗼𝗹𝗶𝗴𝗼𝗻𝘂𝗰𝗹𝗲𝗼𝘁𝗶𝗱𝗲 𝗹𝗶𝗴𝗮𝘁𝗶𝗼𝗻   1️⃣Firstly, so-called '͟𝘀͟𝘁͟𝗶͟𝗰͟𝗸͟𝘆͟ 𝗲͟𝗻͟𝗱͟'͟ 𝗹͟𝗶͟𝗴͟𝗮͟𝘁͟𝗶͟𝗼͟𝗻͟ involves joining of hybridized oligonucleotide fragments that have complementary single-stranded overhangs (sticky ends) at their termini.   Sticky end ligation is highly adaptable and can be performed with different numbers of fragments of various lengths depending on the process and strategy. It is particularly well-suited for the synthesis of siRNA.   👉 For siRNA, Hongene's strategy is to ligate four fragments (2 for each strand), then purify by non-denaturing chromatography purification. This obviates the requirement for separate purification of each siRNA strand and simplifies the manufacturing process.   2️⃣Secondly, oligonucleotides can be synthesized by 𝘀͟𝗽͟𝗹͟𝗶͟𝗻͟𝘁͟𝗲͟𝗱͟ 𝗹͟𝗶͟𝗴͟𝗮͟𝘁͟𝗶͟𝗼͟𝗻͟ where multiple oligonucleotide fragments and corresponding reverse complement splints are hybridized, and then enzymatically ligated.   Splinted ligation is modular and with the appropriate design of fragments and templates can be applied to synthesize oligonucleotides including sgRNA and long prime editing guide RNA (pegRNA) with high purity and yield.   👉For sgRNA Hongene's strategy involves the use of DNA splints which are convenient to produce and highly effective. Following ligation of hybridized oligonucleotide fragments, the splints can be removed by chromatography under denaturing conditions or enzymatically under the action of DNase I to furnish the pure sgRNA.   Check out our on-demand video to learn more about our experience in using ligation to synthesize siRNA and sgRNA. https://lnkd.in/ehU7T5un #oligonucleotides #RNA #siRNA #sgRNA #RNAtherapeutics  #geneediting #chemoenzymaticligation #oligo #drugdevelopment

Alnylam Pharmaceuticals just announced that the FDA has accepted for review supplemental New Drug Application (sNDA) for vutrisiran, an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM). Vutrisiran is the generic name for AMVUTTRA®, which the FDA currently approves for treating the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. By rapidly knocking down both mutant and wild‑type transthyretin (TTR), vutrisiran addresses the underlying cause of transthyretin amyloidosis (ATTR). If approved, Vutrisiran would become the first therapeutic approved in the U.S. to treat both the polyneuropathy manifestations of hATTR and cardiomyopathy manifestations of ATTR amyloidosis. Congratulations to the Alnylam Pharmaceuticals team 👏 ! #RNAi #RNAtherapeutics #amyloidosis #cardiomyopathy

Congratulations to the BridgeBio team Neil Kumar🎉! FDA has announced the approval of Attruby™ (acoramidis), a near complete TTR stabilizer (≥90%) for the treatment of adults with transthyretin amyloid cardiomyopathy (ATTR-CM) to reduce cardiovascular death and cardiovascular-related hospitalization. ATTR-CM is a rare and progressive disease where a protein called transthyretin (TTR) misfolds and forms harmful amyloid deposits in the heart. Over time, these deposits can lead to heart failure, making it difficult for the heart to pump blood properly. Although treatments for ATTR-CM have been limited, the approval of Attruby offers new hope for patients affected by this debilitating disease. The approval of acoramidis comes less than 10 months after BridgeBio Pharma announced the FDA’s acceptance of their New Drug Application (NDA) in February 2024. Attruby is the first and only approved product with a label specifying near-complete stabilization of TTR. Attruby has been shown to preserve the native function of TTR as a transport protein of thyroxine and vitamin A and to demonstrate benefits on cardiovascular outcomes, offering a new treatment option for those affected by this serious disease. #geneticdiseases #cardiomyopathy #rarediseases #clinicaltrial #amyloidosis #biomarker

📢 Researchers from UC San Diego have revealed the pathway leading to protein aggregation in the cytoplasm of motor neurons in individuals with amyotrophic lateral sclerosis (ALS). ALS is a progressive #neurodegenerativedisease characterized by the progressive loss of motor neurons. The buildup of TDP-43, a nucleus-bound gene-expression regulatory protein, is a sign of ALS. The aggregation of TDP-43 in the cytoplasm is caused by abnormal nuclear accumulation of charged multivesicular body protein 7 (CHMP7). In this current study, researchers investigated the possible triggers for CHMP7 aggregation in the nucleus. They used a CRISPR-based microRaft followed by gRNA identification (CRaft-ID) to discover 55 RNA-binding proteins that influence CHMP7 localization. Specifically, they identified an RNA-splicing associated protein called SmD1. They employed IP-MS and enhanced CLIP analyses to reveal CHMP7’s interactions with SmD1. When inhibited, SmD1 significantly increased CHMP7 aggregation in the nucleus. Boosting SmD1 expression kept CHMP7 in the cytoplasm, preserving nucleoporins and keeping TDP-43 in the nucleus for proper gene regulation. Since SMN dysfunction is implicated in spinal muscular dystrophy, and therapeutics like risdiplam exist for this disorder, the researchers are exploring whether risdiplam could be repurposed to treat ALS by enhancing the SMN complex and preventing CHMP7 nuclear buildup. The study suggests that the SMN complex plays a crucial role in the onset of ALS, paving the way for repurposing existing medications or developing new therapeutics for ALS. Congratulations to all contributors🎉 Gene Yeo Norah Al-Azzam, PhD Image credit to original article: https://lnkd.in/epdXq95p #RNA #neurobiology #RNAtherapeutics #ALS #neurodegenerative #geneedtiting

Chemoenzymatic ligation is a promising approach for oligonucleotide and siRNA API manufacture, offering enhanced purity, increased yield, and reduced environmental impact. This innovative technology provides an optimal solution to meet the anticipated surge in demand for siRNA drugs over the coming decade. 🧬𝗪𝗲 𝘂𝘀𝗲 𝘁𝘄𝗼 𝗿𝗼𝗯𝘂𝘀𝘁 𝗺𝗲𝘁𝗵𝗼𝗱𝘀 𝗼𝗳 𝗼𝗹𝗶𝗴𝗼𝗻𝘂𝗰𝗹𝗲𝗼𝘁𝗶𝗱𝗲 𝗹𝗶𝗴𝗮𝘁𝗶𝗼𝗻   1️⃣Firstly, so-called '͟𝘀͟𝘁͟𝗶͟𝗰͟𝗸͟𝘆͟ 𝗲͟𝗻͟𝗱͟'͟ 𝗹͟𝗶͟𝗴͟𝗮͟𝘁͟𝗶͟𝗼͟𝗻͟ involves joining of hybridized oligonucleotide fragments that have complementary single-stranded overhangs (sticky ends) at their termini.   Sticky end ligation is highly adaptable and can be performed with different numbers of fragments of various lengths depending on the process and strategy. It is particularly well-suited for the synthesis of siRNA.   👉 For siRNA, Hongene's strategy is to ligate four fragments (2 for each strand), then purify by non-denaturing chromatography purification. This obviates the requirement for separate purification of each siRNA strand and simplifies the manufacturing process.   2️⃣Secondly, oligonucleotides can be synthesized by 𝘀͟𝗽͟𝗹͟𝗶͟𝗻͟𝘁͟𝗲͟𝗱͟ 𝗹͟𝗶͟𝗴͟𝗮͟𝘁͟𝗶͟𝗼͟𝗻͟ where multiple oligonucleotide fragments and corresponding reverse complement splints are hybridized, and then enzymatically ligated.   Splinted ligation is modular and with the appropriate design of fragments and templates can be applied to synthesize oligonucleotides including sgRNA and long prime editing guide RNA (pegRNA) with high purity and yield.   👉For sgRNA Hongene's strategy involves the use DNA splints which are convenient to produce and highly effective. Following ligation of hybridized oligonucleotide fragments, the splints can be removed by chromatography under denaturing conditions or enzymatically under the action of DNase I to furnish the pure sgRNA.   Check out our on-demand video to learn more about our experience in using ligation to synthesize siRNA and sgRNA. https://lnkd.in/ehU7T5un #oligonucleotides #RNA #siRNA #sgRNA #RNAtherapeutics  #geneediting #chemoenzymaticligation #oligo #sam

Researchers at the University of Rochester Medical Center developed a ribozyme-activated mRNA trans-ligation technique that enables the delivery of large genes for treating muscular dystrophies that require the replacement of large proteins. Muscular dystrophies are genetic disorders characterized by muscle weakness and degeneration. Treatment remains a challenge as traditional vectors, such as AAVs, are limited in their ability to carry large genetic payloads. To address this issue, researchers developed StitchR, which utilizes ribozymes to facilitate the trans-ligation of mRNA fragments. The method involves designing two mRNA fragments that can be efficiently delivered into cells using conventional vectors. Each fragment is equipped with a ribozyme that can cleave and ligate the fragments to form a full-length, functional mRNA molecule. Results demonstrated that StitchR could restore the expression of large functional muscle proteins, such as dysferlin and dystrophin, to near endogenous levels. This method opens new avenues for the efficient delivery of large therapeutic genes, potentially leading to more effective and widely applicable treatments. Congratulations to all contributors🎉! Sean Lindley Kadiam C Venkata Subbaiah Douglas M. Anderson Find out more: https://lnkd.in/e9JNkS2S #DMD #musculardystrophy #RNAtherapuetics #genetherapy #drugdelivey #geneticdiseases

Friday Great News! We are thrilled to be awarded "𝟮𝟬𝟮𝟰 𝗤𝘂𝗮𝗹𝗶𝘁𝘆 𝗪𝗶𝗻𝗻𝗲𝗿" at Nitto Business Summit 2024🎉! Our very own SVP for global BD KS Chin accepted the award on behalf of Hongene at the summit. 🌟A special "Thank You" to Nitto EMEA Nitto Avecia for recognizing our efforts and providing a platform fostering growth and innovation. We are honored to be a part of your community! Asmara Agha Charlie Waserstein 🤝 To our valued customers, your trust and support in Hongene have been the driving force behind our success. Continuing the momentum, we commit to maintaining the highest quality standards in all our endeavors to assist you with excellence! #qualityaward #oligonucleotides #RNAtherapeutics #nucleicacid #chemicalmanufacturing

#breakingnews The FDA just granted accelerated approval to PTC Therapeutics, Inc. gene therapy for the treatment of AADC deficiency, marking the first-ever gene therapy approved in the United States that is directly administered to the brain!🎉 AADC deficiency is a fatal, rare genetic disorder that typically causes severe disability and suffering from the first months of life, leading to decreased muscle tone, movement disorders, and disruption of the autonomic nervous system. This gene therapy, with the brand name KEBILIDI™, is a gene replacement therapy that is directly administered to the putamen of the brain through a stereotactic neurosurgical procedure. The efficacy and safety of KEBILIDI have been demonstrated across clinical trials and compassionate use programs. A previous 5-year analysis showed that pediatric patients with AADC deficiency when treated with KEBILIDI experienced durable developmental, motor, and cognitive improvements. Find out more at https://lnkd.in/eEsgjEXD #AADCdeficiency #genetherapy #rarediseases #neurologicdiseases #geneticdisorder Image credit: https://lnkd.in/eSnftu44