Fixx Pharmaceuticals Wants to Dominate Hemophilia Market With Gene Editing

Prnewswire | August 28, 2020

Fixx Pharmaceuticals Inc. is pleased to announce the launch of its biotechnology business operations in Las Vegas, Nevada and San Francisco, California. The company is focused on developing a cure for Hemophilia through the use of CRISPR gene editing. There are currently 20,000 people in the U.S. who suffer from hemophilia and 400,000 globally. "We are looking to commercialize a product as soon as possible," says Jesse John Francis Clark, founder of Fixx. "We are going to develop a product that is more cost-effective for payers. We're looking to keep it under $2 million per patient," he said, saying that the overall cost of care of hemophilia patients will decrease. "If we are successful in developing a commercialized product, we are looking at upwards of $40 billion in sales from the U.S. market alone. I think our investors will be happy with that," says Clark.

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GSK beat fourth-quarter profit and sales forecasts helped by sales of its shingles vaccine and unveiled an upbeat forecast for 2023.

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GSK beat fourth-quarter profit and sales forecasts helped by sales of its shingles vaccine and unveiled an upbeat forecast for 2023.

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CONTINUUS Pharmaceuticals Adopts Digital Validation

CONTINUUS Pharmaceuticals | March 15, 2023

On March 14, 2023, ValGenesis, Inc., a leading enterprise validation lifecycle management solutions provider, announced that CONTINUUS Pharmaceuticals has successfully implemented the ValGenesis Validation Lifecycle Management System (VLMS) for efficient management of validation processes across the company in a 100% digital format. CONTINUUS Pharmaceuticals is a rapidly growing small business specializing in end-to-end integrated continuous manufacturing (ICM) of pharmaceuticals. As a spin-out company resulting from a long-term collaboration between MIT and Novartis, CONTINUUS Pharmaceuticals is committed to enhancing the accessibility and affordability of pharmaceutical products worldwide by exploring advanced manufacturing paradigms that expedite the development of raw materials and APIs to final dosage forms. Due to the immense validation requirements involved in the production of different drug products, CONTINUUS Pharmaceuticals needed an electronic solution that determines the disposition of their assets while switching amongst product lines. By leveraging the ValGenesis VLMS, CONTINUUS Pharmaceuticals has successfully implemented facilities, equipment validation, computer system and utility validation. The ValGenesis VLMS is the system of record for CONTINUUS Pharmaceuticals' engineering team and will be expanded to other teams in the future. The highly automated, real-time, and compliant system provided by ValGenesis minimizes the potential for Good Manufacturing Practice (GMP) non-compliance while accelerating time to market. About CONTINUUS Pharmaceuticals CONTINUUS Pharmaceuticals is an end-to-end integrated continuous manufacturing (ICM) company specializing in pharmaceutical production. The company is committed to enhancing the accessibility and affordability of pharmaceutical products worldwide by exploring advanced manufacturing paradigms that expedite the development of APIs and raw materials to final dosage forms. It leverages a novel continuous manufacturing platform called Integrated Continuous Manufacturing (ICM), initially developed through a $65 million joint research project between Novartis and MIT. With ICM, it aims to transform and streamline the supply chain of pharmaceuticals by delivering high-quality, lower-cost drugs "on-demand."

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BUSINESS INSIGHTS, PHARMACY MARKET

Caris Life Sciences and Xencor Expand Target Discovery Collaboration

Caris Life Sciences and Xencor | January 06, 2023

Caris Life Sciences®, a molecular science and technology company, and Xencor, Inc., a clinical-stage biopharmaceutical company, have announced that they will work more closely together to research, develop, and sell new bispecific and multispecific XmAb® antibodies. This expanded relationship will boost the number of targets and cancer types that Caris will investigate with Caris Discovery, a proprietary multi-omics discovery engine platform. The foundation for drug candidate development on Xencor's XmAb platform will be novel targets and their accompanying validation packages. Under the terms of the new contract, Xencor will receive worldwide commercial rights to study, develop, and market products aimed at up to three targets discovered through collaboration. In addition, Caris will get an upfront payment and may receive up to $187 million in commercial milestone payments and development. The company will also be eligible for royalties on the net sales of products commercialized as a result of the agreement, as well as future rights for molecular profiling and companion diagnostics. Caris Discovery leverages the collective force of its unmatched platform by combining the data generated from the primary patient tissues' molecular interrogation using ADAPT™, which integrates the company's extensive catalog of molecular and clinical data. About Caris Life Sciences Caris Life Sciences® is a Texas-based molecular science firm revolutionizing healthcare. Its market-leading molecular profiling products analyze DNA, RNA, and proteins to produce a molecular blueprint. This plan shows how patients, doctors, and researchers can use the information to improve outcomes and save lives. The company also uses cutting-edge algorithms for artificial intelligence (AI) and machine learning (ML) to improve precision medicine. About Xencor Xencor develops modified antibodies and cytokines for cancer and autoimmune illnesses. More than 20 XmAb® candidates are in clinical development while three are sold by partners. The company’s XmAb technology modifies protein structures to create new therapeutic pathways.

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Harrow Introduces Next-Gen Compounded Atropine Formulations

Harrow | February 16, 2023

On February 15, 2023, Harrow, a renowned pharmaceutical firm specializing in eyecare in the United States, announced the launch of its next-generation, patent-pending compounded Atropine formulations. These formulations are now accessible through ImprimisRx, a mail-order pharmacy subsidiary wholly owned by Harrow. The following are the key features of ImprimisRx's next-generation Atropine formulations Thoroughly validated analytical tests are conducted to ensure potency, consistency, and stability. The formulations are free of preservatives and boric acid. They possess a physiologic pH of 5.5. They can be stored at room temperature for up to 180 days. Eyecare professionals can purchase these compounded Atropine formulations for as less as $39 per 5mL bottle through various channels, such as online via atropine.com or by contacting ImprimisRx Customer Service at 615-437-4690 via call or text. Compounded formulations are recommended for patients with clinical requirements that are not met by FDA-approved medications. It is essential to note that compounded medications are not reviewed by FDA for their safety and effectiveness. ImprimisRx does not manufacture copies of products that are already available in the market. Harrow's Chairman and Chief Executive Officer, Mark L. Baum, commented, "Our mission has always been to help our eyecare professionals address their patients' unmet needs and to make ophthalmic medications both accessible and affordable. The availability of these innovative compounded atropine formulations is in keeping with our mission. We believe ophthalmologists, optometrists, and the millions of Americans who may benefit from topical atropine therapy, will appreciate the tremendous value these patent-pending formulations offer." (Source – Business Wire) About Harrow Founded in 2012, Harrow is a leading ophthalmic pharmaceutical firm in the United States focused on discovering, developing, and introducing accessible, innovative, and affordable prescription therapies. It holds commercial rights for ten FDA-approved eyecare pharmaceutical products. In addition, the company also operates ImprimisRx, a leading U.S. compounding business that specializes in ophthalmic medications and is licensed to ship prescription drugs in all 50 states. The success of its efforts is reflected in its remarkable financial performance, with a 72% compound annual growth rate achieved during our first eight years in operation. This phenomenal growth rate has established Harrow as one of the fastest-growing ophthalmic pharmaceutical companies in the United States.

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