INDUSTRIAL IMPACT, MEDICAL
prnewswire | April 28, 2023
Hansa Biopharma AB, "Hansa" a pioneer in enzyme technology for rare immunological conditions, and Genethon, a pioneer and a leader in gene therapy research and development for rare genetic diseases, announced they have entered a research and development collaboration.
The collaboration will, in a clinical study, evaluate the safety and efficacy of Hansa's antibody cleaving enzyme imlifidase as a pre-treatment prior to the administration of Genethon's gene therapy product candidate GNT-0003 in Crigler-Najjar syndrome in patients with pre-existing neutralizing antibodies (NAbs) to adeno-associated virus serotype 8 (AAV8). The presence of circulating NAbs today excludes patients from entering clinical studies with potentially curative gene therapy treatments and from future access to approved gene therapies.
Søren Tulstrup, President and CEO, Hansa Biopharma stated: "Genethon is a pioneer at the cutting-edge of research and development of gene therapies for rare diseases and we are thrilled to be collaborating with them. This research collaboration further validates Hansa's commitment in gene therapy and underscores the important role that our antibody-cleaving enzyme technology can play in ensuring that even more patients can benefit from life-saving gene therapies".
GNT-0003 is currently being evaluated in a pivotal clinical study in France, Italy, and the Netherlands and has received PRIME (PRIority MEdicines) status from the EMA. Through the collaboration announced today, patients with Crigler-Najjar and pre-formed antibodies to AAV8 will be enrolled in a study with similar design where imlifidase is evaluated as a pre-treatment to enable gene therapy treatment with GNT-0003. The outcome of the ongoing clinical study of GNT-0003 could potentially form the basis for a MAA or BLA application in Europe or the US.
Crigler-Najjar is a genetic disease-causing bilirubin accumulation which leads to irreversible neurological damage manifested as muscle weakness, lethargy, deafness, mental retardation, and eye movement paralysis. Crigler-Najjar syndrome is an ultra-rare disease affecting less than one case per one million people per year.1
Frédéric Revah, CEO, Genethon added: "Patients with pre-existing neutralizing antibodies against AAV vectors cannot today benefit from gene therapy. This collaboration with Hansa Biopharma is thus an important next step in the development of our gene therapy treatment for Crigler-Najjar syndrome, Hansa Biopharma's proven enzyme technology coupled with its scientific expertise will help us advance the critical research we are conducting in Crigler-Najjar and could enable gene therapy treatment for patients who are today not eligible because of their immunological status".
About Hansa Biopharma
Hansa Biopharma is a pioneering commercial-stage biopharmaceutical company on a mission to develop and commercialize innovative, lifesaving and life-altering treatments for patients with rare immunological conditions. Hansa has developed a first-in-class immunoglobulin G (IgG) antibody-cleaving enzyme therapy, which has been shown to enable kidney transplantation in highly sensitized patients. Hansa has a rich and expanding research and development program based on the Company's proprietary IgG-cleaving enzyme technology platform, to address serious unmet medical needs in transplantation, autoimmune diseases, gene therapy and cancer. Hansa Biopharma is based in Lund, Sweden, and has operations in Europe and the U.S. The Company is listed on Nasdaq Stockholm under the ticker HNSA.
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.
Globenewswire | May 19, 2023
Mustang Bio, Inc. a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for difficult-to-treat cancers and rare genetic diseases, today announced a strategic update, including anticipated milestones for 2023. Mustang intends to optimize the allocation of its resources and focus on MB-106, MB-109, and in vivo CAR T platform technology. Additionally, Mustang announced a partnership with uBriGene (Boston) Biosciences Inc. (“uBriGene”), the U.S. subsidiary of uBriGene Group, a leading cell and gene therapy contract development and manufacturing organization (“CDMO”), which includes the sale of the Company’s development, manufacturing and analytical testing facility in Worcester, Massachusetts to uBriGene.
Under the terms of an asset purchase agreement between Mustang and uBriGene, uBriGene will acquire Mustang’s state-of-the-art clinical- and commercial-scale cell and gene therapy manufacturing facility in Worcester, Massachusetts, for a total consideration of $11 million. This consideration includes $6 million payable upfront plus an additional $5 million payable upon Mustang raising $10 million in gross proceeds from equity raises following the closing of the transaction. The closing of the transaction is subject to the satisfaction of certain conditions, including approval of transfer of the Company’s lease to uBriGene by the owner of the building (an affiliate of the University of Massachusetts Chan Medical School) and the acceptance of offers of employment with uBriGene or its affiliates by certain key current Mustang employees. Subject to satisfaction of conditions, the Company expects the transactions to close in June 2023.
Subject to closing, the parties will enter into a manufacturing supply agreement, under which uBriGene will manufacture Mustang’s lead product candidates, including continuing to support MB-106 manufacturing for the ongoing multi-center Phase 1/2 trial.
Mustang’s Worcester facility is a 27,000 square foot, cutting edge cGMP facility supporting process development, manufacturing and analytical testing, designed with the flexibility to expand and support various cell and gene therapy production requirements and capacities. uBriGene intends to expand the Worcester site’s capabilities while leveraging Mustang’s experienced staff and robust quality and operating systems to manufacture a broader portfolio of advanced modalities. uBriGene will also offer their expertise in preclinical research services and late-stage and commercial manufacturing of advanced therapy products with respect to product and process characterization, and regulatory inspections.
Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, commented, “We are very pleased to have found a great partner for the manufacturing of our CAR T cell and gene therapies, and we believe that this strategic partnership with uBriGene will meet our portfolio manufacturing needs to reach critical upcoming data inflection points, while extending our cash runway. I want to thank our manufacturing team for their dedication in building and growing our Worcester facility since it opened in 2018. While we are optimizing our resources at Mustang, we look forward to continuing to work with many of our colleagues in this new capacity, as our CDMO.”
“This acquisition is important to uBriGene’s commitment to support the development, clinical, and commercial supply of cell and gene therapies to sustain industry demand and provide new CDMO options,” said Alex Chen, President of uBriGene. “We look forward to working together with the University of Massachusetts Chan Medical School and local biotechnology companies to continue to advance the manufacturing ecosystem in the Greater Boston region. This partnership enables us to expand rapidly to create a North American presence and offer the same high-quality cell and gene therapy development and manufacturing capabilities for the U.S. that we currently provide in Asia, including to support Mustang Bio’s lead clinical-stage CAR-T program.”
Mustang Bio Strategic Portfolio Updates
CAR T Cell Therapies
After a review of its portfolio of product candidates to determine the future strategy of its programs and the proper allocation of its resources, Mustang will discontinue development of its MB-102, its CD123-targeted CAR T cell therapy, as well as its HER2-, CS1- and PSCA-targeted CAR T cell therapy programs, comprising a portion of the Company’s portfolio of CAR T cell therapies being developed by the Company in partnership with City of Hope.
Mustang will continue to work with Fred Hutchinson Cancer Center to develop MB-106 and with Mayo Clinic to develop its in vivo CAR T platform technology. Mustang will also continue to work with City of Hope and with Nationwide Children’s Hospital on the development of MB-109 (MB-101 CAR T cell therapy targeting IL13Rα2 on malignant glioma cells + MB-108 oncolytic virus to potentially make these tumors more susceptible to killing by the CAR-T cells).
Additionally, based on a review of the data from the investigator-sponsored clinical trials of the gene therapy for X-linked severe combined immunodeficiency (“XSCID”) that has been licensed to Mustang Bio, enrollment to these trials has been paused. We await data from new investigator-sponsored trials being planned by our partners that will test a modified version of the current lentiviral vector prior to initiating multicenter Mustang-sponsored trials in both the newborn and previously transplanted patient populations. No safety concerns in the trials utilizing the current vector have been noted to date and no insertional mutagenesis or malignancy has been detected in either of the two investigator-sponsored trials. However, Mustang has made the decision to delay initiating its own sponsored trials out of an abundance of caution, and once we have had the opportunity to review the emerging data from the planned trials utilizing the modified vector, Mustang expects to provide more information on timelines
About Mustang Bio
Mustang Bio, Inc. is a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to outlicense or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR-T therapies across multiple cancers, as well as lentiviral gene therapies for severe combined immunodeficiency. Mustang’s common stock is registered under the Securities Exchange Act of 1934, as amended, and Mustang files periodic reports with the U.S. Securities and Exchange Commission (“SEC”). Mustang was founded by Fortress Biotech, Inc.