INDUSTRIAL IMPACT, MEDICAL
Globenewswire | March 27, 2023
Iovance Biotherapeutics, Inc. a late-stage biotechnology company developing novel T cell-based cancer immunotherapies, announced it has completed its rolling Biologics License Application (BLA) submission to the U.S. Food and Drug Administration (FDA) for lifileucel. Lifileucel is a tumor infiltrating lymphocyte (TIL) therapy intended as a treatment for patients with advanced melanoma who progressed on or after prior anti-PD-1/L1 therapy and targeted therapy, where applicable. There are no FDA approved therapies in this treatment setting.
Frederick Vogt, Ph.D., J.D., Interim President and Chief Executive Officer of Iovance, stated, “Completing our BLA submission for lifileucel is a critical step forward in our journey to deliver the first individualized, one-time cell therapy for a solid tumor. I would like to acknowledge the patients and physicians who participated in the C-144-01 clinical trial and the FDA review team for their commitment and support, as well as our internal team for their tremendous effort in completing the first BLA submission for Iovance. Our preparations for commercialization remain on track to support a launch later this year. We look forward to continued collaboration with the FDA as they review this new class of treatment for advanced melanoma patients with limited options.”
The BLA submission for lifileucel is supported by positive clinical data from the C-144-01 clinical trial in patients with advanced post-anti-PD1 melanoma. Following a successful pre-BLA meeting with the FDA, Iovance is pursuing accelerated approval in this indication. Iovance also reached agreement with the FDA regarding the registrational trial design for the Phase 3 TILVANCE-301 trial of lifileucel in combination with pembrolizumab in frontline advanced melanoma. TILVANCE-301 is intended to support full approval of lifileucel in post-anti-PD-1 advanced melanoma and is also designed to support registration for lifileucel in combination with pembrolizumab as therapy for advanced melanoma in the frontline setting. Startup activities for TILVANCE-301 are ongoing and the trial is expected to be well underway at the time of potential accelerated approval for lifileucel in advanced post-anti-PD-1 melanoma.
Marc Hurlbert, Ph.D., CEO of the Melanoma Research Alliance (MRA), said, “MRA congratulates Iovance for completing the BLA submission and moving closer toward making TIL therapy an option for people with advanced melanoma who have progressed following prior treatments. We hope for an FDA approval as quickly as possible for patients with significant unmet need who have no approved treatment options.”
Following receipt of the complete rolling BLA submission for lifileucel, the FDA has 60 days to determine the acceptability of the BLA for review. The rolling BLA allowed Iovance to submit portions of the BLA to the FDA on an ongoing basis, enabling the FDA to begin review as early as possible as documents were received. The rolling BLA submission and eligibility for priority review are benefits available under the FDA’s guidance on expedited programs for serious conditions, which allow for an expedited six-month review from the time of BLA acceptance. In addition, the FDA previously granted a regenerative medicine advanced therapy (RMAT) designation for lifileucel in advanced melanoma.
About Iovance Biotherapeutics, Inc.
Iovance Biotherapeutics aims to be the global leader in innovating, developing and delivering tumor infiltrating lymphocyte (TIL) therapies for patients with cancer. We are pioneering a transformational approach to cure cancer by harnessing the human immune system’s ability to recognize and destroy diverse cancer cells in each patient. Our lead late-stage TIL product candidate, lifileucel for metastatic melanoma, has the potential to become the first approved one-time cell therapy for a solid tumor cancer. The Iovance TIL platform has demonstrated promising clinical data across multiple solid tumors. We are committed to continuous innovation in cell therapy, including gene-edited cell therapy, that may extend and improve life for patients with cancer.
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MEDTECH, MEDICAL
Businesswire | May 23, 2023
Twist Bioscience Corporation a company enabling customers to succeed through its offering of high-quality synthetic DNA using its silicon platform, today announced the launch of a portfolio of RNA sequencing tools, which includes the Twist RNA Exome, Twist RNA Library Prep Kit and the Twist Ribosomal RNA (rRNA) & Hemoglobin (Globin) Depletion Kit, as well as custom target enrichment capabilities for RNA and whole transcriptome sequencing. This comprehensive suite of RNA tools can enable targeted or whole transcriptome research across fields including precision medicine, biomarker discovery and immuno-oncology research.
“Twist’s RNA sequencing portfolio builds on our established NGS capabilities in genomic sequencing and methylation detection and expands into gene expression analysis, opening a new and significant market for Twist,” said Emily M. Leproust, Ph.D., CEO and co-founder of Twist Bioscience. “This offering allows our customers to leverage Twist’s leading target enrichment platform to discover and resolve low abundance RNA transcripts in the human transcriptome. Our RNA sequencing workflows are designed specifically for challenging samples. By enriching relevant transcripts, we can enable researchers to get high-quality data from a range of sample types, including damaged RNA.”
Unlike the genome, which is relatively static, the transcriptome changes over time in response to varying disease states and drug therapies. Gene expression levels also differ across tissues and cell types. To fully understand this dynamic quality of RNA, researchers often sequence RNA samples from the same source at multiple time points, incurring additional costs and resource burdens. Each component of the Twist RNA sequencing portfolio is purpose-built to maximize data quality, minimize workflow inefficiency, decrease wasted sequencing reads, and support comprehensive transcriptomic profiling across even low-quality and low-input samples common in oncology.
The Twist RNA sequencing portfolio provides end-to-end workflows that can be used to study total RNA and measure the whole transcriptome, protein coding sequences of messenger RNA (mRNA) or custom targets. These products can also be integrated into customers’ existing workflows. Twist RNA sequencing tools enable minimal hands-on time and increase the scale of samples sequenced through utilizing automation. They are compatible with a wide range of sample inputs, including difficult or low-quality samples. RNA panel designs can also now incorporate Twist’s new exon-aware design algorithm, which reduces bias in detection and can provide an important tool for researchers studying rare gene transcripts that drive tumor biology.
Twist Targeted RNA Sequencing Workflow
Twist target enrichment for RNA leverages the performance, efficiency and sensitivity of Twist target enrichment for sequencing of RNA transcripts of interest across samples and species. It can be used to enrich relevant transcripts with the RNA Exome or in custom RNA panels.
Twist RNA Exome
The Twist RNA Exome with Twist’s exon-aware design algorithm enables targeted sequencing of the protein coding regions of the human transcriptome without bias toward specific combinations of exons. The Twist RNA Exome covers up to 99.9% of protein coding sequencing in Gencode and RefSeq databases. This coverage, along with the exon-aware design approach, could enable researchers to capture important transcripts including those in low input and degraded samples from formalin fixed paraffin embedded (FFPE) sections.
Twist Whole Transcriptome Sequencing Workflow
Twist Whole Transcriptome Sequencing can be used to discover novel transcripts, including low expressing genes and novel isoforms. It can be used with a variety of samples including whole blood, fresh and frozen tissue samples and low quality samples, such as FFPE samples. The Twist rRNA & Globin Depletion Kit can be used with this workflow to deplete rRNA and hemoglobin targets so that researchers can study the rest of the transcriptome. The full workflow can be completed in less than five hours with minimal hands-on time.
About Twist Bioscience Corporation
Twist Bioscience is a leading and rapidly growing synthetic biology and genomics company that has developed a disruptive DNA synthesis platform to industrialize the engineering of biology. The core of the platform is a proprietary technology that pioneers a new method of manufacturing synthetic DNA by “writing” DNA on a silicon chip. Twist is leveraging its unique technology to manufacture a broad range of synthetic DNA-based products, including synthetic genes, tools for next-generation sequencing (NGS) preparation, and antibody libraries for drug discovery and development. Twist is also pursuing longer-term opportunities in digital data storage in DNA and biologics drug discovery. Twist makes products for use across many industries including healthcare, industrial chemicals, agriculture and academic research.
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CELL AND GENE THERAPY, INDUSTRY OUTLOOK
Globenewswire | May 10, 2023
Adaptive Biotechnologies Corporation a commercial stage biotechnology company that aims to translate the genetics of the adaptive immune system into clinical products to detect and treat disease, announced that the U.S. Food and Drug Administration has accepted an investigational new drug (IND) application submitted by its collaborator, Genentech, a member of the Roche Group, for a T-cell receptor (TCR) based T-Cell Therapy. This is the first TCR-based therapeutic product candidate to advance into clinical development based on Adaptive’s collaboration with Genentech in oncology.
“This IND acceptance reaffirms the value of our immune medicine platform and Adaptive’s ability to identify and characterize clinical grade, therapeutic T-cell receptors, which is the cornerstone of our drug discovery capabilities,” said Chad Robins, chief executive officer and co-founder, Adaptive Biotechnologies. “We look forward to supporting Genentech’s world-class team of scientists and drug developers to advance this potentially life-saving therapy into the clinic for patients with solid tumors.”
Under the terms of Adaptive and Genentech’s collaboration agreement, Genentech has responsibility for clinical, regulatory and commercialization efforts for any T-Cell Therapy product candidate. Adaptive reiterates its full year revenue guidance in the range of $205 to $215 million.
About Adaptive Biotechnologies
Adaptive Biotechnologies is a commercial-stage biotechnology company focused on harnessing the inherent biology of the adaptive immune system to transform the diagnosis and treatment of disease. We believe the adaptive immune system is nature’s most finely tuned diagnostic and therapeutic for most diseases, but the inability to decode it has prevented the medical community from fully leveraging its capabilities. Our proprietary immune medicine platform reveals and translates the massive genetics of the adaptive immune system with scale, precision and speed. We apply our platform to partner with biopharmaceutical companies, inform drug development, and develop clinical diagnostics across our two business areas: Minimal Residual Disease (MRD) and Immune Medicine. Our commercial products and clinical pipeline enable the diagnosis, monitoring, and treatment of diseases such as cancer, autoimmune disorders, and infectious diseases. Our goal is to develop and commercialize immune-driven clinical products tailored to each individual patient.
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