INDUSTRIAL IMPACT, MEDICAL
Molecular Assemblies, Inc. | March 15, 2023
On March 14, 2023, Molecular Assemblies, Inc., a leading life sciences company, made a significant announcement regarding its enzymatic DNA synthesis technology. The company has successfully shipped the first enzymatically synthesized oligonucleotides to leading researchers in biotechnology, academic, and synthetic biology. The capacity to synthesize lengthy, extremely pure DNA promptly utilizing a proprietary Fully Enzymatic Synthesis (FES) technology solves critical unmet demands in DNA synthesis, thus allowing demanding and high-value applications such as CRISPR gene editing.
Long, highly pure single-stranded oligonucleotides are critical in various applications, such as CRISPR gene editing tools used in therapeutic development, research discovery and protein engineering. However, the availability of desired sequences required to make advanced edits has been a significant bottleneck.
Molecular Assemblies' Key Client Program gives chosen researchers priority access to lengthy and customized oligonucleotides synthesized utilizing the company's FES technology. Thanks to this initiative, customers are expected to speed up their research in CRISPR gene editing and other applications needing long oligonucleotides. In addition, key customers can give input to shape the future of enzymatic DNA synthesis. Molecular Assemblies plans to present more details about the technology at the SynBioBeta 2023 conference in Oakland, California, from May 23-25.
Molecular Assemblies' President and CEO Michael J. Kamdar commented, "With the achievement of this significant commercial milestone, we are proving that fully enzymatic synthesis can deliver the DNA that customers want today, without compromise," He further emphasized, "Customers want application-ready DNA, unrestricted by length and sequence-complexity, which we know is essential to power new breakthroughs in scientific research and accelerate innovation for many industries, including next generation medicines and technologies."
(Source – PR Newswire)
About Molecular Assemblies, Inc.
Molecular Assemblies, Inc. is a leading company dedicated to developing enzymatic DNA synthesis technology to address key challenges in DNA synthesis. Its proprietary Fully Enzymatic Synthesis (FES) technology aims to provide an innovative solution to produce long, pure DNA quickly, efficiently, and sustainably. As a result, molecular Assemblies' FES technology has the potential to transform the field of DNA synthesis by enabling the production of high-quality, long oligonucleotides. This development has significant implications for research discovery, therapeutic development, and protein engineering, particularly in the context of CRISPR gene editing. The company was founded in 2013 and is headquartered in San Diego, California. It has received significant funding from various sources, including venture capital firms, angel investors, and government agencies.
CELL AND GENE THERAPY, AI
Businesswire | June 05, 2023
BenevolentAI, a leader in the development of cutting-edge AI that accelerates biopharma discovery, announces the successful delivery of its pre-clinical candidate for the potential treatment of amyotrophic lateral sclerosis (ALS), BEN-34712.
BEN-34712 is an oral, potent and selective brain penetrant RARɑβ (retinoic acid receptor alpha beta) biased agonist and will now enter investigational new drug (IND)-enabling studies.
Impaired retinoic acid signalling has been shown to result in neuroinflammation, oxidative stress and mitochondrial dysfunction, all hallmarks of ALS. In preclinical studies conducted by the Company, BEN-34712 was neuroprotective in a patient-derived, disease-relevant in vitro motor neuron/iAstrocyte co-culture model, demonstrating significant efficacy in both sporadic and familial subtypes of ALS. In addition, BEN-34712 has demonstrated both central nervous system (CNS) target engagement and functional protective effects in the SOD1G93A mouse model of ALS after 50-day repeat dosing.
BenevolentAI collaborated with the Sheffield Institute for Translational Neuroscience (SITraN) at the University of Sheffield on this programme, utilising their patient-derived motor neuron/iAstrocyte co-culture systems and in vivo model expertise.
Anne Phelan, Chief Scientific Officer, BenevolentAI, said: “There remains a significant and urgent need for new and alternative therapies for patients with ALS. We are pleased by the promising advancement of our drug candidate, BEN-34712, towards clinical development, backed by the compelling preclinical data generated by our collaborators at SITraN.”
Richard Mead, Senior Lecturer in Translational Neuroscience at SITraN, commented: "ALS patients suffering from this devastating neurodegenerative disease are in dire need of effective therapy, with the current standard of care options focusing on symptom management or offering limited clinical benefit. We believe BEN-34712 represents an exciting development in our research for a potential new treatment, particularly as it shows effectiveness in both the SOD1G93A mouse model system as well as familial and C9orf72 related ALS patient-derived cell models."
BenevolentAI is a leading developer of advanced artificial intelligence technologies that unlock the value of multimodal data, surface novel insights, and accelerate biomedical discovery. Through the combined capabilities of its AI platform, its scientific expertise, and wet-lab facilities, the Company is developing an in-house drug pipeline of high-value assets. The Company is headquartered in London, with a research facility in Cambridge (UK) and a further office in New York.
ALS is a progressive neurologic disorder characterised by the loss of cortical and spinal motor neurons, leading to the denervation of nerve endplates, axonal retraction and subsequent muscle atrophy. The average survival time following the initial diagnosis is around two-three years, and while there are drugs approved by the US FDA for ALS, they provide only modest benefits to patients, underwriting the urgent need for new and alternative therapies.
About SITraN at the University of Sheffield
The Sheffield Institute for Translational Neuroscience (SITraN) is an essential development in the fight against motor neurone disease and other common neurodegenerative disorders, including Parkinson's and dementia, as well as stroke and multiple sclerosis. SITraN has the potential to bring new treatments and new hope to patients and carers in the UK and worldwide, by significantly accelerating the pace of therapeutic development using technologies such as experimental modelling of disease, gene therapy and stem cell biology, gene expression profiling and bioinformatics analysis and modelling of the biological processes. Since its opening by Queen Elizabeth II in 2010, SITraN has grown immensely and developed into a leading global facility which is at the forefront of research and expertise.
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.