Francis Mojica, the Spanish Scientist Who Discovered CRISPR

labiotech.eu | April 08, 2019

Contrary to popular belief, Francis Mojica was the first person to discover the CRISPR mechanism and give it its name. He told me how the discovery came about, discussed the wide potential of the gene editing tool and shared his favorite CRISPR application. You’ve likely heard of CRISPR/Cas9, a gene editing tool that has taken the world by storm. Although it’s far from being the first tool to edit DNA, CRISPR technology makes gene editing much easier and faster than before. In the last few years, its use has become ubiquitous in labs around the world. CRISPR/Cas9 is used to speed up drug discovery and the creation of animal models to study all sorts of diseases. But the application everyone is avidly expecting and throwing money at is using CRISPR to cure human diseases. The first clinical trials have already started, and their results will reveal whether CRISPR can fulfill these big expectations.

Spotlight

If you're wondering how the Drug Supply Chain Security Act's 2023 requirements will affect your pharmaceutical lab, then now might be a great time to take a look at Zebra Technologies' fixed industrial scanners and machine vision.

Spotlight

If you're wondering how the Drug Supply Chain Security Act's 2023 requirements will affect your pharmaceutical lab, then now might be a great time to take a look at Zebra Technologies' fixed industrial scanners and machine vision.

Related News

BUSINESS INSIGHTS, PHARMA TECH

Neochromosome Announces Successful Collaboration with Sumitovant Biopharma

Sumitovant Biopharma, Inc. | January 20, 2023

On January 19, 2023, Neochromosome, Inc., announced a partnership with Sumitovant Biopharma, Inc., a technology-driven biopharmaceutical firm expediting the discovery and commercialization of novel therapeutics. This collaboration blends Neochromosome's neoYeast™ platform with Sumitovant's DrugOME™ computational ecosystem to identify new proteins that would benefit from non-canonical amino acid chemistries that are unavailable in nature. As part of the collaboration, Sumitovant used a combination of natural language processing (NLP), artificial intelligence (AI), machine learning (ML), and computational algorithms to identify opportunities for Neochromosome to enhance the discovery and production of proteins limited by evolution and traditional fabrication processes. For example, non-canonical amino acid inclusion allows new-to-nature proteins with improved structures and functions but often requires chemical synthesis, which is expensive, size- and complexity-restricted, and environmentally damaging. neoYeast™, based on Neochromosome's world-leading genome-scale cell engineering technology stack, enables the highly efficient and environmentally friendly integration of non-canonical amino acids into proteins, with both discovery and manufacturing possible in a single genome-engineered yeast strain. Sumitovant used DrugOME to explore libraries of traditional and non-traditional sources of data to recommend targeted protein development candidates for neoYeast. Co-Founder and CEO of Neochromosome, Leslie Mitchell, commented, "The combination of Sumitovant's technology platform and life-sciences expertise with Neochromosome's ability to both discover and biomanufacture novel proteins in a single system enables Neochromosome to accelerate and streamline the delivery of better therapies for patients." She added, "We are excited to partner with the world-class team at Sumitovant to identify opportunities to create better versions of existing drugs, along with new-to-nature compounds, and to manufacture them efficiently at scale." (Source – Business Wire) Sumitovant Biopharma, Inc. The firm has supported the development of multiple FDA-approved products and a strong pipeline of early-through late-stage investigational assets addressing unmet needs in pediatrics, oncology, urology, women's health, specialty respiratory, and infectious diseases through its proprietary computing and data platforms, scientific expertise, and diverse company portfolio. It is a wholly-owned subsidiary of Sumitomo Pharma.

Read More

PHARMA TECH

Xeris Biopharma Announces Research Collaboration and Option Agreement With Horizon Therapeutics plc for XeriJect™ Formulation of Teprotumumab

Xeris | November 24, 2022

Xeris Biopharma Holdings, Inc. a growth-oriented biopharmaceutical company committed to improving patient lives by developing and commercializing innovative products across a range of therapies, announced that it has entered into a research collaboration and option agreement with Horizon Therapeutics plc. Under the terms of the agreement, Xeris will use its proprietary formulation technology platform, XeriJect™, to develop an ultra-concentrated, ready-to-use, subcutaneous injection of teprotumumab and Horizon will have an option to license the Xeris technology. Teprotumumab is the first and only medicine approved by the U.S. Food and Drug Administration for the treatment of Thyroid Eye Disease a serious, progressive and potentially vision-threatening rare autoimmune disease. Teprotumumab-trbw is known as TEPEZZA® in the United States. Xeris will receive an upfront payment, and may be entitled to receive development milestones, regulatory milestones, and salesbased milestones, as well as royalties based on future sales if the commercial license option is exercised. Specific financial terms of the agreement were not disclosed. “We are excited to announce our collaboration with Horizon for the development of a subcutaneous formulation of teprotumumab using our XeriJect technology to potentially enhance the patient experience and delivery of the treatment for Thyroid Eye Disease. This partnership demonstrates the potential value of our technology to enable large molecule subcutaneous injections that provide a more patient friendly regimen that is effective, safe, and more convenient, with potential for improved adherence.” Paul R. Edick, Chairman and CEO of Xeris About XeriJect™ XeriJect formulations are innovative, ready-to-use, viscoelastic pharmaceutical suspensions that have the potential to improve drug delivery, lower treatment burden and improve patients' lives across a broad range of therapeutic categories. XeriJect suspensions maximize drug loadings at >400mg/mL, enable small volume subcutaneous injections and do not settle on storage. The suspensions use FDA-approved excipients and leverage known manufacturing processes. XeriJect formulation technology is well suited for drugs and biologics including large molecules such as proteins, monoclonal antibodies, and vaccines. The technology is protected by an extensive patent estate, trade secrets and know-how, and it is available for licensing. About Xeris Xeris is a growth-oriented biopharmaceutical company committed to improving patients’ lives by developing and commercializing innovative products across a range of therapies. Xeris has three commercially available products; Gvoke®, a ready-to-use liquid glucagon for the treatment of severe hypoglycemia, Keveyis®, the first and only FDA-approved therapy for primary periodic paralysis, and Recorlev® for the treatment of endogenous Cushing’s syndrome. Xeris also has a robust pipeline of development programs to extend the current marketed products into important new indications and uses and bring new products forward using its proprietary formulation technology platforms, XeriSol™ and XeriJect™, supporting long-term product development and commercial success.

Read More

PHARMACY MARKET

TC BioPharm Begins Dosing Phase 2B Clinical Study Evaluating its Lead Compound, OmnImmune®, in Patients with Acute Myeloid Leukemia

TC BioPharm | November 23, 2022

TC Biopharm a clinical stage biotechnology company developing platform allogeneic gamma-delta T cell therapies for cancer today announced the dosing of its first three patients within its Phase 2b clinical trial of OmnImmune®, an allogeneic unmodified cell therapy focused on treating Acute Myeloid Leukemia. The initial 5 patients in the trial are deemed a "safety cohort", spaced two weeks apart with safety review by an oversight board to confirm no drug related toxicity issues, subsequent to 5 patients being dosed the study will advance to open enrollment. This safety cohort is in line with TCBP's step-wise clinical trial advancement, moving from donor matching in the Phase 1b to a universal donor model with no HLA matching of donor to patient. "The launch of our Phase 2B trial is a key milestone in the development of our lead therapeutic, OmnImmune®, for patients with AML and for TC BioPharm's emerging pipeline of 'off-the-shelf' gamma-delta T cell therapies. This study design includes a 5 patient safety cohort prior to open enrollment, we expect to complete the safety cohort before the end of 2022. The next step in the study is a 19 patient interim review, which will allow TCBP to review dosing and increase dosing to a higher level should our team deem it necessary for efficacy, or we can elect to maintain our current dosing level of 7x10^7 or 700 million cells per dose. We look forward to moving ahead with our Phase 2b trial with a target for open enrollment in January 2023, as well as our efforts to expand our clinical efforts in the US in the first half of 2023." Bryan Kobel, CEO of TC BioPharm TC BioPharm's Phase 2B trial, dubbed ACHIEVE, will enroll adults diagnosed with AML who have either relapsed or are refractory to prior treatments as well as a cohort for patients with myelodysplastic syndromes conditions that can occur when the blood-forming cells in the bone marrow become abnormal. The trial is expected to enroll approximately 37 patients. About TC BioPharm PLC TC BioPharm is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of gamma-delta T cell therapies for the treatment of cancer with human efficacy data in acute myeloid leukemia. Gamma-delta T cells are naturally occurring immune cells that embody properties of both the innate and adaptive immune systems and can intrinsically differentiate between healthy and diseased tissue. TC BioPharm uses an allogeneic approach in both unmodified and CAR modified gamma-delta T cells to effectively identify, target and eradicate both liquid and solid tumors in cancer. TC BioPharm is the leader in developing gamma-delta T cell therapies, and the first company to conduct phase II/pivotal clinical studies in oncology. The Company is conducting two investigator-initiated clinical trials for its unmodified gamma-delta T cell product line - Phase 2b/3 pivotal trial for OmnImmune® in treatment of acute myeloid leukemia using the Company's proprietary allogenic CryoTC technology to provide frozen product to clinics worldwide. TC BioPharm also maintains a robust pipeline for future indications in solid tumors as well as a significant IP/patent portfolio in the use of CARs with gamma-delta T cells and owns our manufacturing facility to maintain cost and product quality controls.

Read More