INDUSTRIAL IMPACT, MEDICAL
Prnewswire | April 11, 2023
Pharming Group N.V. announces the first commercial shipments of Joenja® to patients in the United States. Joenja®, an oral, selective PI3Kδ inhibitor, is the first and only treatment approved in the U.S. for activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS), a rare and progressive primary immunodeficiency, in adult and pediatric patients 12 years of age and older.
Under the terms of Pharming's 2019 exclusive license agreement with Novartis for leniolisib, the corresponding first commercial sale of Joenja® triggers a $10 million milestone payment by Pharming to Novartis.
Stephen Toor, Chief Commercial Officer of Pharming, commented
"We are pleased to announce that the first Joenja® shipments to patients, with payor reimbursement, were delivered approximately two weeks following FDA approval, achieving an important milestone for patients suffering with APDS. We look forward to making Joenja® widely available across the U.S. as the first and only approved treatment for patients with APDS."
About Activated Phosphoinositide 3-Kinase δ Syndrome (APDS)
APDS is a rare primary immunodeficiency that was first characterized in 2013. APDS is caused by variants in either one of two identified genes known as PIK3CD or PIK3R1, which are vital to the development and function of immune cells in the body. Variants of these genes lead to hyperactivity of the PI3Kδ (phosphoinositide 3-kinase delta) pathway, which causes immune cells to fail to mature and function properly, leading to immunodeficiency and dysregulation.1,2,3 APDS is characterized by a variety of symptoms, including severe, recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy.4,5 Because these symptoms can be associated with a variety of conditions, including other primary immunodeficiencies, it has been reported that people with APDS are frequently misdiagnosed and suffer a median 7-year diagnostic delay.6 As APDS is a progressive disease, this delay may lead to an accumulation of damage over time, including permanent lung damage and lymphoma.4-7 A definitive diagnos
is can be made through genetic testing. APDS affects approximately 1 to 2 people per million worldwide.
About Pharming Group N.V.
Pharming Group N.V. is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases. Pharming is commercializing and developing an innovative portfolio of protein replacement therapies and precision medicines, including small molecules, biologics, and gene therapies that are in early to late-stage development. Pharming is headquartered in Leiden, Netherlands, and has employees around the globe who serve patients in over 30 markets in North America, Europe, the Middle East, Africa, and Asia-Pacific.
INDUSTRIAL IMPACT, MEDICAL
Twist Bioscience | March 10, 2023
On March 9, 2023, Twist Bioscience Corporation, a leading firm providing high-quality synthetic DNA through its silicon platform, announced the launch of integrated antibody discovery services. This premium offering combines the company's synthetic libraries and AI machine learning with an in vivo immunization approach acquired through its acquisition of Abveris, also known as Twist Boston. This service provides customers with optimized, development-ready antibody candidates.
The combined team, now called Twist Biopharma Solutions, is led by Tracey Mullen, MBA, the senior vice president of biopharma for Twist Bioscience. This newly formed entity leverages years of industry expertise, scientific excellence, and proprietary technology to discover the best antibody candidate for therapeutic targets of interest.
Twist Biopharma Solutions offers an end-to-end workflow for centralized antibody discovery and optimization, including target validation, hit generation, lead selection, lead optimization, and lead characterization. Twist, supported by high-throughput DNA synthesis and IgG antibody production, constructs extensive and particular synthetic antibody libraries with discovery starting with either in vivo or in vitro diversity.
The newly integrated in vivo discovery approach from Abveris of single B cell screening and hybridoma discovery enables parallel paths where multiple technology methods can be leveraged to build a panel of highly diverse antibody leads with broad epitope coverage. In addition, this multi-pronged approach can increase the likelihood of discovering highly specific, high-affinity functional antibodies by sampling synthetic and natural diversity.
The South San Francisco and Boston biopharma teams were combined to create Twist Biopharma Solutions, led by Tracey Mullen. Aaron Sato, Ph.D., remains integrally involved in Twist Biopharma Solutions and, as a chief scientific officer of Twist Bioscience, extends his expertise across the organization. Tracey Mullen joined Twist Bioscience in November 2021 through the acquisition of Abveris. In April 2022, she took on the position of senior vice president of operations, driving the completion of the Factory of the Future in Wilsonville, Oregon, which began shipping products in January 2023. Before joining Twist, she served as CEO of Abveris and worked on the antibody discovery team at Biogen.
About Twist Bioscience
Twist Bioscience is a leading biotechnology company specializing in synthetic biology and genomics. It has developed a proprietary DNA synthesis platform that enables the production of custom-designed DNA strands faster, more accurately, and at a lower cost than traditional methods. In addition, Twist collaborates with partners across industries to develop customized DNA sequences for specific applications and offers a range of other products and services, such as oligonucleotides, synthetic genes, and custom panels for next-generation sequencing. The company has also developed a library of over 100 billion synthetic antibodies, which can be used for diagnostic and therapeutic applications.
CELL AND GENE THERAPY, INDUSTRIAL IMPACT
Globenewswire | May 02, 2023
Editas Medicine, Inc. a clinical stage genome editing company, announced that the journal Nature Biotechnology published the comprehensive data from a study of the proprietary SLEEK gene editing technology.
Despite major progress in achieving gene disruption with CRISPR-Cas gene editing technologies, efficient knock-in of transgenes continues to be a significant challenge for the gene editing field. To solve this challenge, SLEEK was developed to enable high knock-in efficiency with both viral and non-viral transgene formats while also ensuring robust simultaneous expression of up to four transgene cargos.
The study demonstrated that utilizing SLEEK results in the knock-in of multiple clinically relevant transgenes through a proprietary process that specifically selects only those cells containing the knock-in cargo. This process was developed by leveraging Editas Medicine’s proprietary engineered AsCas12a nuclease, which can achieve very high editing efficiency while maintaining high specificity. More than 90 percent knock-in efficiencies were observed in various clinically relevant target cells, including T cells, B cells, iPSCs, and NK cells. Additionally, SLEEK can be used to fine-tune the expression levels of transgene cargos, an important feature of next-generation cell therapies. As a demonstration of SLEEK’s potential value in clinical applications, the study authors used SLEEK to generate iPSC-derived NK cells capable of high-levels of in vivo persistence and robust tumor clearance in a solid tumor animal model.
“We are thrilled Nature Biotechnology published our paper sharing the comprehensive data on our SLEEK gene editing technology as we believe SLEEK has immense potential for gene editing drug development. As shared in the publication, SLEEK technology enables nearly 100 percent knock-in of functional transgene cargos at specific locations in the genome which may result in highly efficient multi-transgene knock-in for the next generation of cell therapy medicines,” said John A. Zuris, Ph.D., Director of Editing Technologies, Editas Medicine, and senior author on the study.
Editas Medicine believes that SLEEK may enable better product purity as well as shorter manufacturing timelines for the next generation of cell therapy medicines. Earlier this year, the Company announced it licensed its interest in the SLEEK technology to Shoreline Biosciences for specific usage in iPSC-derived NK and iPSC-derived macrophage cell therapies for oncology. The SLEEK technology remains an important Editas capability in iPSC engineering for a wide variety of future applications.
About Editas Medicine
As a clinical stage genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cas12a genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of Broad Institute and Harvard University’s Cas9 patent estates and Broad Institute’s Cas12a patent estate for human medicines.