Feliza Mirasol is the science editor for 

Feliza Mirasol

Myeloid Therapeutics, a US-based clinical-stage messenger RNA (mRNA)-immunotherapy company, announced on May 18, 2023 that investment firm Hatteras Investment Partners has completed a round of financing totaling $73 million, which included participation from new investors, ARCH Venture Partners and Moore Strategic Ventures, as well as existing investors, Newpath Partners, 8VC, and Alexandria Venture Investments.

According to a company press release, proceeds from the financing will support Myeloid’s continued clinical development of MT-101, its lead cell therapy program in Phase I/II studies for T-cell lymphoma. The funding will also accelerate the development of MT-302, a trophoblast cell-surface antigen 2 (TROP2)-FcA mRNA-lipid nanoparticle (LNP) product, into a Phase I/II study for TROP2-expressing tumors. The funding will also help advance additional 

“Myeloid continues to make significant progress across all aspects of our business. We are pioneering the convergence of immunology and RNA science to build a clinical-stage portfolio of products, starting with the significant unmet needs of cancer patients,” said Daniel Getts, CEO of Myeloid, in the press release. “We are pleased that such high-quality investors have joined with us to accelerate this effort.”

“We are highly encouraged by the innovation, execution, and clinical development progress demonstrated by Myeloid. We are proud to support this [company] through its next phase of growth and are optimistic that these approaches will drive a meaningful clinical outcome for patients,” said Clay B. Thorp, general partner at Hatteras, in the release.

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With $73 million in financing from investment firms, Myeloid Therapeutics plans to accelerate its lead clinical candidate into Phase I/II development for treating solid tumors.

Myeloid Therapeutics Gets $73 Million to Advance mRNA-based Pipeline

Sania Therapeutics, a new UK-based startup focused on developing genetic medicines for neural circuit dysfunction, has launched itself from stealth mode at the American Society of Gene & Cell Therapy (ASGCT) conference, held in Los Angeles, Calif., on May 16–20. On May 17, 2023, the company unveiled its suite of proprietary patented platforms at ASGCT.

The company’s technology combines the evolution of adeno-associated viruses (AAVs) in human neural circuits—the fundamental components of the nervous system—with tuneable, conditionally activated ion channels. This combination permits breakthrough efficacy by correcting dysfunctional neural excitation, which unlocks a broad range of diseases both within and beyond neurology, according to a company press release.

The company’s foundational technology platforms were first developed at University College London. The launch at ASGCT follows the closing of a series seed financing. Proceeds from the seed financing have been applied to platform development, the advancement of a lead program in motor disorders, and the initiation of additional undisclosed programs that leverage the company’s platform capabilities.

Sania’s R-Scan platform enables human-centric precision delivery. R-Scan leverages the combination of microfluidics with a diverse population of human-induced pluripotent stem cells to recreate human neural circuits in a dish. This recreation 

 enables directed evolution of human-centric AAVs. R-Scan is complemented by a second platform, called Gre-Scan, which is a novel and proprietary technology for the high throughput screening of gene regulatory elements.

Both R-Scan and Gre-Scan are combined with Neu-Scan, which facilitates tuneable genetic neuromodulation via testing and validation of the overexpression of conditionally activated ion channels. The company’s proof-of-concept data, which were presented at ASGCT, demonstrate that the Neu-Scan platform has identified ion channels, deliverable by AAV, that enable the treatment of dysfunctional neural excitation.

“We formed Sania with a simple yet powerful vision: to be the first in developing medicines that treat disorders of neural circuits selectively. Today we are excited to demonstrate proof-of-principle data and unveil our platforms,” said Andy Murray, CEO and co-founder of Sania Therapeutics, in the press release.

Sania Therapeutics has emerged from stealth mode and launched its technology platforms for enabling the development of novel therapeutics to treat neural circuit dysfunction.

Launch of Sania Therapeutics Expected to Advance Development of Novel Therapeutics for Neural Circuit Dysfunction

Gilead Sciences and Arcus Biosciences, a US-based clinical-stage biopharmaceutical company, announced on May 15, 2023 that they are expanding a previously announced research collaboration to now include development of therapies for treating inflammatory diseases. Their initial collaboration agreement, worth potentially more than $1.6 billion, focused on oncology (1).

Under the expanded collaboration, Arcus will receive an upfront payment of $35 million. Arcus will initiate research programs against up to four targets that are jointly selected by both parties and that are applicable to inflammatory diseases. Gilead would have the option to license each program at two separate, prespecified time points. If Gilead exercises its option for the first two target programs at the earlier time point, then Arcus would be eligible to receive up to $420 million in option and milestone payments as well as tiered royalties for each optioned program. For any other option exercised by Gilead for the four target programs, the two parties would have rights to co-develop the candidates. In addition, both parties would share global development costs and co-commercialize and share profits in the United States for optioned programs.

“We are pleased to build upon Gilead’s efforts in inflammation with the expansion of our strategic collaboration with Arcus,” said Flavius Martin, executive vice-president, Research, Gilead Sciences, in a company press release. “Arcus is an excellent partner with clear strengths in discovery and development, and we believe this partnership will significantly accelerate our progress in developing transformative new therapies for inflammatory diseases.”

“Since its founding, Arcus has been creating and developing therapeutic interventions that can modulate the immune system to treat cancer. Through this expansion of our partnership with Gilead, we can combine our research expertise in immunology and small-molecule drug discovery with Gilead’s strong clinical experience in inflammation,” said Terry Rosen, CEO, Arcus, in the release. “The research collaboration facilitates much earlier alignment between Gilead and Arcus on our discovery and development activities, while enabling Arcus to expand into inflammation in a capital-efficient manner.”

1. Gilead Sciences, Gilead Sciences and Arcus Biosciences Establish 10-year Partnership to Co-develop and Co-commercialize Next-generation Cancer Immunotherapies. Press Release, May 27, 2020.

Gilead Sciences and Arcus Biosciences have expanded their research collaboration to include inflammatory diseases in addition to oncology. 

Gilead Sciences and Arcus Biosciences Expand Research Partnership

On May 15, 2023, the Canadian Advanced Therapies Training Institute (CATTI) announced that it has launched a cell and gene therapy (CGT) training center of excellence at the University of Guelph in Guelph, Ontario, Canada through a partnership with the university, CCRM, a Canada-based public–private partnership, and OmniaBio, a subsidiary of CCRM and a contract development and manufacturing organization specializing in the production of gene-modified cells and viral vectors. According to the institute’s press release, the new center is CATTI’s first in-person training site at the university.

The CGT training program is slated to begin in the summer of 2023 and will cater to recent post-secondary graduates, industry professionals, and companies looking to grow their cleanroom manufacturing staff. The site will begin to address the industry’s critical training needs, and, according to CATTI in the press release, the program represents the first stage of a larger, multi-site training strategy for the institute.

In its first year, the program will focus on human pluripotent stem cell banking, aseptic techniques, good manufacturing practices (GMPs) optimized for CGT manufacturing, and lab skills, including pipetting, vessel handling, microscope operation, and other skills. The curriculum is expected to include a combination of online courses and hands-on courses in a specialized training lab. The lab is designed to focus on providing GMP industry standard skill sets, according to the press release.

“The biosafety level 2 lab space at the University of Guelph will expand CATTI’s offering in central Canada and provide hands-on-learning in a one-of-a-kind, state-of-the-art space for workers across Canada and internationally,” said Vanessa Laflamme, CEO, CATTI, in the release. “We expect to train [more than] 600 people in the next two years, including [more than] 300 employees for OmniaBio, one of CATTI’s strategic customers.”

“OmniaBio has a significant need for trained talent due to the growth trajectory and technical capability of its operation,” said Mitchel Sivilotti, CEO, OmniaBio, in the release. “This collaboration with CATTI and the University of Guelph is a win for everyone involved. We will support the effort by providing funding, strategic guidance, and content development input to facilitate a hands-on, highly-focused, GMP training experience utilizing the most current equipment and technologies used in CGT manufacturing today.”

OmniaBio, which was itself recently launched by CCRM in March 2022 (1), is also in the process of constructing new facilities. In the first phase of construction, OmniaBio is building an 89,000-ft2 (7,432-m2) facility, which will be equipped with 15 clean rooms and employ 500 staff members. This first facility is expected to be completed in 2024. A second building is also planned. Upon completion, OmniaBio and CCRM will have more than 50 cleanrooms and more than 1000 employees.

1. CCRM, Funding from Ontario Government Launches OmniaBio Inc. Press Release, March 31, 2022.

Canadian Advanced Therapies Training Institute

CATTI has launched an in-person training site at the University of Guelph in Ontario, Canada, for training personnel in cell and gene therapy manufacturing.

CATTI Starts Up CGT Training Center at University of Guelph

On May 15, 2023, BiOneCure Therapeutics, a US-based clinical-stage biopharmaceutical company focused on developing antibody-drug conjugates (ADCs), announced that it has entered into a strategic collaboration with China- based Nanjing Leads Biolabs, a clinical-stage biopharmaceutical company focused on second-generation immuno-oncology therapeutics, to develop a range of innovative ADCs for treating solid tumors.

The companies intend to leverage their complementary technologies in the collaboration, which they expect will strengthen their R&D and product development capabilities. BiOneCure’s technology includes several proprietary ADC payload-linker platforms. Its first product, BIO-106, is an anti-Trop-2 ADC that is currently in a Phase I clinical study. Leads Biolabs’ portfolio holds more than 20 novel mono- or bispecific antibody drug projects to fulfill unmet medical needs, according to a company press release.

"We are excited to collaborate with Leads Biolabs to develop ADC drugs," said Haifeng Bao, CEO of BiOneCure, in the press release. "Through this collaboration, we aim to accelerate innovation and development in the ADC field and provide better treatment options for patients."

"We believe that BiOneCure has leading ADC technology platforms, and [its] ADC molecule is in the clinical stage," said Xiaoqiang Kang, founder and chairman of Leads Biolabs, in the release. "Leads Biolabs is a clinical-stage antibody new drug company, with multiple antibody technology platforms. We are convinced that this collaboration will greatly expand the innovative R&D capabilities of both parties."

Under the collaboration, BiOneCure Therapeutics and Nanjing Leads Biolabs intend to develop a range of innovative antibody-drug conjugates for treating solid tumors.

BiOneCure Therapeutics and Nanjing Leads Biolabs Partner to Develop ADCs for Solid Tumors

Synthego, a US-based provider of genome engineering solutions, has opened its new good manufacturing practice (GMP) synthesis facility. According to a May 11, 2023 company press release, the 18,000-ft

 facility will support the growing demand for the development of high-quality, clustered regularly interspaced short palindromic repeats (CRISPR)-enabled 

 therapeutics. Services at the new facility will also include investigational new drug-, new drug application-, and biologics license application-enabling clinical studies.

The new facility features multiple multiplexed manufacturing lines, fully automated controls, and an integrated manufacturing execution system. The company will have the capability to produce a range of oligonucleotides, including synthetic single guide RNAs. The facility also includes dedicated process development, analytical testing, and quality control areas, which will ensure that all aspects of the manufacturing process are closely controlled. The facility meets the requirements of both domestic and international regulatory authorities including FDA, the European Medicines Agency, the Medicines and Healthcare products Regulatory Agency, and Health Canada, according to the press release.

“This facility will be essential in supporting the burgeoning field of clinical phase, first-in-human, CRISPR-enabled medicines both for more common genetic diseases but also for rare diseases affecting children for whom other treatments do not exist,” said Adrian Thrasher, a clinician and professor of Pediatric Immunology at University College London Great Ormond Street Institute of Child Health, in the press release. Thrasher’s laboratory is developing new CRISPR-enabled medicines for children with rare genetic diseases.

“As we move into this new generation of cell and gene therapies, it will be crucial that there are strong, reliable manufacturing capabilities that exist for the necessary components of the CRISPR technology—such as the guide RNA—in order to accelerate the path to clinic and treat patients," Thrasher said in the release.

“Synthego pioneered the mass production of guide RNAs for discovery research that has enabled many of the CRISPR-based cell and gene therapy trials happening today. Having been a key leader in providing the technology for researchers and clinicians, it follows that they are now expanding their capacity to support this research as it translates into the clinic,” said Don Kohn, distinguished professor and clinician of Pediatric Hematology and Oncology at University of California, Los Angeles. Kohn’s laboratory pioneered genetic therapies for immunocompromised children.

Synthego has opened its new GMP-compliant RNA manufacturing facility in Redwood City, Calif., for CRISPR-enabled genomics therapeutics.