Francis Mojica, the Spanish Scientist Who Discovered CRISPR

labiotech.eu | April 08, 2019

Contrary to popular belief, Francis Mojica was the first person to discover the CRISPR mechanism and give it its name. He told me how the discovery came about, discussed the wide potential of the gene editing tool and shared his favorite CRISPR application. You’ve likely heard of CRISPR/Cas9, a gene editing tool that has taken the world by storm. Although it’s far from being the first tool to edit DNA, CRISPR technology makes gene editing much easier and faster than before. In the last few years, its use has become ubiquitous in labs around the world. CRISPR/Cas9 is used to speed up drug discovery and the creation of animal models to study all sorts of diseases. But the application everyone is avidly expecting and throwing money at is using CRISPR to cure human diseases. The first clinical trials have already started, and their results will reveal whether CRISPR can fulfill these big expectations.

Spotlight

Merck & Co, known as MSD outside of the United States and Canada, is a global pharmaceutical company whose mission is to discover, develop and provide innovative products and services that save and improve lives around the world.

Spotlight

Merck & Co, known as MSD outside of the United States and Canada, is a global pharmaceutical company whose mission is to discover, develop and provide innovative products and services that save and improve lives around the world.

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BUSINESS INSIGHTS, PHARMA TECH

Rallybio Corporation and EyePoint Announce Research Cooperation

EyePoint Pharmaceuticals | February 28, 2023

On February 27, 2023, EyePoint Pharmaceuticals, Inc., a leading pharmaceutical firm committed to the development and commercialization of therapeutics to help improve the lives of patients with severe eye disorders and Rallybio Corporation, a clinical-stage biotechnology company dedicated to identifying and accelerating the development of life-transforming therapeutics for patients with severe and rare diseases, announced a research collaboration to explore the sustained delivery of Rallybio's complement component 5 (C5) inhibitor for the treatment of geographic atrophy, an advanced form of age-related macular degeneration that can cause irreversible vision loss. The research will use EyePoint's proprietary Durasert® technology, which is designed for sustained intraocular drug delivery. Approximately one million individuals in the United States suffer from geographic atrophy, which is characterized by atrophic lesions in the central region of the macula. Geographic atrophy can cause irreversible vision loss and can lead to legal blindness. Currently, geographic atrophy treatments are limited in choice and duration of action, leaving a significant unmet need for safe and effective treatment options. Under the terms of the research partnership, EyePoint and Rallybio will assess and explore the viability of utilizing Rallybio's C5 inhibitor in EyePoint's Durasert technology, with the intention to increase the collaboration upon mutual agreement following the evaluation. EyePoint Pharmaceuticals' M.D., President and Chief Operating Officer Jay Duker commented, "We are excited to begin this research collaboration to explore the combination of Rallybio's C5 inhibitor with our bioerodible Durasert sustained release drug delivery technology to develop a potential long-acting treatment for geographic atrophy," He further emphasized, "Geographic atrophy associated with dry macular degeneration is a devastating eye disease, and the inhibition of complement is a proven treatment pathway. We hope to leverage our Durasert technology in this collaboration to create a potential best-in-class, long-acting intravitreal insert, which we believe could provide a more desirable option for patients given that the existing approved therapy is injected every one to two months." (Source – Business Wire) About EyePoint Pharmaceuticals EyePoint Pharmaceuticals is a leading pharmaceutical company that develops and commercializes therapeutic products to help patients suffering from serious eye disorders. Headquartered in Watertown, Massachusetts, it is committed to using its innovative drug delivery platform to improve patients' quality of life. The company's pipeline utilizes its proprietary Durasert technology, which enables sustained intraocular drug delivery. Its current pipeline includes EYP-1901, a potential six-month intravitreal anti-VEGF treatment aimed at treating wet age-related macular degeneration. Durasert technology has been successfully used across four FDA-approved products, including YUTIQ®, which treats chronic non-infectious uveitis affecting the posterior segment of the eye.

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BUSINESS INSIGHTS, PHARMACY MARKET

Accord Healthcare Expands Offerings with Generic Relapsing MS Drug

Accord Healthcare | March 14, 2023

On March 13, 2023, Accord Healthcare informed that it has expanded its portfolio with Teriflunomide, a film-coated tablet equivalent to Sanofi Genzyme's Aubagio. The drug treats relapsing forms of MS in adults, including clinically-isolated syndrome and active secondary progressive disease. The company offers Teriflunomide in 14 mg and 7 mg tablets, now available for shipping. Teriflunomide has been granted FDA approval as an immunomodulatory agent for patients aged 18 and above. Its purpose is to reduce the frequency and severity of relapses, which can lead to new or worsening neurological symptoms associated with the disease. Accord Healthcare US Vice President of Sales Jim Brown stated, "We're proud to add Teriflunomide to our portfolio. This is an important oral therapy for the millions of patients living with relapsing MS." He added, "Generic medicine allows broad access to effective therapy, to help improve disease course and potentially slow progression of MS." (Source – Cision PR newswire) Teriflunomide is an immunomodulatory medication that inhibits an enzyme critical to a metabolic pathway that supports lymphocyte proliferation. This results in fewer activated lymphocytes, which minimizes inflammation and immunological reactivity, resulting in lower recurrence rates and protection of brain volume against inflammatory nerve cell attacks. Around 2.8 million people globally have multiple sclerosis, with the majority diagnosed with relapsing-remitting MS that typically begins in their twenties or thirties. Diagnosis involves experiencing two or more attacks or relapses while maintaining stability. New or exacerbated symptoms characterize relapses, and the immune system attacks the myelin sheath surrounding nerve fibers, ultimately causing long-term damage. Symptoms include numbness, fatigue, double vision, and balance problems. Besides, relapsing-remitting MS can also progress to active secondary progressive disease. About Accord Healthcare Accord Healthcare, a subsidiary of Intas Pharmaceuticals, is recognized as a prominent player in the generic pharmaceutical industry. The company's vision is to enhance patient access to medicines and revolutionize the pharmaceutical experience for healthcare providers and patients. With an emphasis on advanced manufacturing technology and in-house research, Accord Healthcare delivers a wide range of affordable, complex, and essential medicines. The company's dedication to forward-thinking and innovative solutions remains at the core of its mission. Headquartered in Durham (North Carolina), its products are marketed in 80 countries through the Intas network.

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PHARMACY MARKET, PHARMA TECH

Merck & XtalPi Collaboration Optimizes Drug Formulations with AI-Powered Techniques

prnewswire | April 18, 2023

Science and technology company Merck and XtalPi Inc., a pioneering pharmaceutical technology company powered by artificial intelligence (AI) and automation, have jointly published a study that highlights the benefits of combining computational workflows with wet lab experiments to advance drug development. Traditional methods like milling and micronization, used to address undesirable crystal morphologies, can be disruptive and costly. In contrast, molecular dynamics simulations offer valuable insights for better crystallization experiment designs. The collaboration between Merck and XtalPi focused on the impact of different polymer additives on the crystal habit of metformin HCl, a diabetes medication. By combining Merck's experimental capabilities with XtalPi's morphology prediction platform, a comprehensive screening approach for crystal morphology engineering was developed. XtalPi's custom-made force fields successfully predicted the influence of polymer additives on metformin HCl's crystal habits. The addition of HPMC led to a transformation from a needle-like to a prismatic morphology, in agreement with experimental observations. The publication demonstrates the potential of merging computational methods and experimental expertise for drug formulation optimization. Dr. Peiyu Zhang, XtalPi's Chief Scientific Officer, said, "By integrating XtalPi's molecular dynamics predictions with Merck's experimental validation, we have taken a step towards the rational engineering of desirable crystal habits. We hope to continue improving this 'dry + wet lab' method with leading industry experts like Merck, in the production of novel therapeutics worldwide." Dr. Jan Gerit Brandenburg, Head of Digital Chemistry at Merck, added, "Our collaboration with XtalPi is transforming pharmaceutical development. By seamlessly incorporating computer simulations with our experimental formulation expertise in a 'digital-first' approach, we are boosting drug development processes and positively impacting patients' lives." About XtalPi XtalPi is a quantum physics and AI-powered pharmaceutical technology platform that provides integrated drug discovery solutions for global pharmaceutical companies. XtalPi has pioneered a new model for drug R&D that leverages a tightly interwoven trifecta of AI, autonomous labs, and expert domain knowledge. The company applies this three-pronged approach in its R&D platform to accelerate the drug discovery and development process and address unmet needs for patients worldwide.

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