CELL AND GENE THERAPY, INDUSTRIAL IMPACT
Bio-Techne Corporation | March 20, 2023
Bio-Techne Corporation announced it has completed its participation in the Cell and Gene Therapy Catapult Process Analytical Technology (Catapult PAT) consortium, a multi-year collaboration involving 24 partner companies, technology providers, therapy developers, and charities. The Catapult PAT consortium is the first initiative of this scale and functional expertise with member companies and organizations collaborating to develop cell and gene therapy-specific process analytical technologies.
The PAT initiative began in January 2021 with a series of experiments designed to monitor process parameters of an exemplar 8-day T-cell expansion bioprocess using primary T-cells. Data were gathered from five independent analyses to generate an extensive, world-first dataset. An analysis of multiple cytokine targets was conducted using high-plex Luminex® immunoassays and the precision multiplexing Ella™ platform from Bio-Techne. The automated workflow of the Ella platform proved to be particularly amenable to the efficiency, accuracy, and throughput demands inherent in a bioprocess manufacturing environment. With its robust performance in this project, the Cell and Gene Therapy Catapult plans to incorporate Ella™ into an existing CGT Catapult facility to further support scientists in the UK.
"As a leader in the forefront of innovative tools to measure cytokines, we are honored to be a part of the Catapult PAT initiative to help advance cell and gene therapy development," said Will Geist, President of Bio-Techne's Protein Sciences Segment. "Bio-Techne remains excited about the potential of these advanced therapies to improve patient outcomes and treat a wide range of diseases that currently have very poor or ineffective treatments."
Established by Innovate UK, the Cell and Gene Therapy Catapult fosters collaborations between academia, industry, and healthcare providers to develop new technology and drive innovation. Catapult experts cover all aspects of advanced therapies, from research and development to clinical adoption and every step in between.
About Bio-Techne
Bio-Techne Corporation is a global life sciences company providing innovative tools and bioactive reagents for the research and clinical diagnostic communities. Bio-Techne products assist scientific investigations into biological processes and the nature and progress of specific diseases. They aid in drug discovery efforts and provide the means for accurate clinical tests and diagnoses. With thousands of products in its portfolio, Bio-Techne generated approximately $1.1 billion in net sales in fiscal 2022 and has approximately 3,000employees worldwide.
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CELL AND GENE THERAPY, INDUSTRIAL IMPACT
Axcella | February 16, 2023
On February 15, 2023, Axcella Therapeutics, a clinical-stage biotechnology firm that develops innovative approaches to complex diseases using multi-targeted endogenous metabolic modulator (EMM) compositions, announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application to initiate a phase 2b/3 trial in the United States for AXA1125 for the treatment of Long COVID Fatigue.
The company stated that it has obtained FDA regulatory guidance in favor of a trial that would serve as the registration trial for patients with Long COVID Fatigue. The trial now has acceptance from both the U.K. and U.S. regulatory authorities.
Last month, the company also announced a regulatory path to registration for the treatment of Long COVID Fatigue with AXA1125, having received regulatory guidance from the U.K.'s regulatory agency, The Medicines and Healthcare products Regulatory Agency (MHRA), supporting a single trial that can serve as the registration trial for patients suffering from Long COVID Fatigue.
The guidance follows the company's submission of data to both regulatory agencies, including results from the Phase 2a randomized, double-blind, placebo-controlled study, which showed that patients who received AXA1125 experienced statistically and clinically significant improvements in both physical (p=0.0097) and mental fatigue (p=0.0097) scores compared to those on the placebo.
Consistent with the Ph2a trial, the trial for phase 2b/3 will register patients with fatigue lasting at least 12 weeks post-COVID-19 infection. The primary endpoint will use the same patient reported outcome tool, the Chalder Fatigue Questionnaire (CFQ-11), to measure fatigue improvements alongside physical function, quality of life, and ability to return to work as additional endpoints. The trial participants will receive AXA1125 or a placebo for three months.
Long COVID is a persistent long-term effect of the pandemic, which affects a large number of patients worldwide, estimated to be around one hundred million. Fatigue is the most frequently reported symptom, with recent estimates indicating that 15-20% of Americans with COVID suffer from ongoing health problems, and up to four million Americans are unable to work because of Long COVID symptoms. In addition, Long COVID has caused approximately $1 trillion in lost earnings and $544 billion in increased medical expenses.
About Axcella
Founded in 2010, Axcella is a clinical-stage biotechnology firm dedicated to introducing a novel approach to treating complex diseases using endogenous metabolic modulator (EMM) compositions. Its product candidates contain EMMs and derivatives that are engineered in distinct ratios and combinations to reset multiple biological pathways, improve cellular energetics, and restore homeostasis. The company's pipeline includes lead therapeutic candidates undergoing Phase 2 development for the reduction in risk of overt hepatic encephalopathy (OHE) recurrence, the treatment of Long COVID, and the treatment of non-alcoholic steatohepatitis (NASH).
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CELL AND GENE THERAPY, INDUSTRIAL IMPACT
Ligand Pharmaceuticals | February 21, 2023
In a recent announcement by biopharmaceutical firm, Ligand Pharmaceuticals, the U.S. Food and Drug Administration (FDA) has given accelerated approval for FILSPARITM (sparsentan). This will be helpful in treating adults with primary IgAN condition who are at risk of rapid disease progression, typically defined as a urine protein-to-creatinine ratio (UPCR) of less than 1.5 g/g.
FILSPARI, a once-daily oral medicine, is the first and only non-immunosuppressive therapy authorized for the treatment of IgAN. Two critical pathways are selectively targeted of IgAN (endothelin-1 and angiotensin II) to check disease progression. Up to 150,000 Americans may have the rare kidney illness IgAN, which is the main cause of kidney failure brought on by glomerular disease. Among these patients, it is predicted that 30,000 to 50,000 can be treated using the indication that has received accelerated approval. Travere has stated that it anticipates FILSPARI to be available from February 27, 2023, and it will offer a thorough patient support program all the way through the patient's therapeutic journey.
Eric Dube, Ph.D., President and CEO of Travere Therapeutics, said, “The accelerated approval of FILSPARI is a significant milestone on our path to advancing a transformative treatment for the IgA nephropathy community.” He further added, “As a first-of-its-kind, non-immunosuppressive therapy, we believe FILSPARI has the potential to ultimately become the new standard of care for IgA nephropathy and offer hope to those living with this condition who until now have had few treatment options. We are grateful to the patients, caregivers, clinical trial investigators, healthcare providers, and advocates who have worked alongside us to develop this innovative first-in-class therapy.”
(Source – Business Wire)
About Ligand Pharmaceuticals
Ligand Pharmaceuticals creates or acquires technologies that aid in the discovery and development of drugs by pharmaceutical firms. The company’s business model generates value for stockholders by offering a diverse portfolio of biotech and pharmaceutical product income streams, that are underpinned by an effective and low corporate cost structure.
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