Andrew Eckert

Children’s National Hospital successfully performed the first-ever high-intensity focused ultrasound (HIFU) surgery on a pediatric patient with neurofibromatosis (NF). This is the youngest patient to undergo HIFU treatment in the world. The advancement of children’s medical devices in the U.S. continues to significantly lag behind adult devices. This is why this milestone marks a significant advance in making pediatric surgery more precise and less invasive.

The hospital is offering this treatment to patients under an ongoing research clinical trial. Children’s National is one of the first pediatric hospitals in the nation to use HIFU for neuro-oncology patients. It’s also the first hospital in the world to use it to treat a pediatric patient with NF. NF is a condition that occurs in approximately 1 in 3,500 births and causes tumors to form in the brain, spinal cord and nerves.

“Using HIFU to treat our pediatric patients is a quantum leap towards non-invasive surgery for kids,” said Robert Keating, M.D., division chief of Neurosurgery and co-director of the HIFU program at Children’s National. “It’s exciting because the future is now here and it’s significantly better for our kids, in terms of non-invasive surgery with lower risk of complications and no exposure to radiation.”

Focused ultrasound (FUS) is a non-invasive therapeutic technology with the potential to transform the treatment of many medical disorders by using ultrasonic thermal energy to specifically target tissue deep in the body. The technology can treat without incisions or the need of radiation.

Click here to read more via Children’s National.

Intima Bioscience to use MaxCyte’s Flow Electroporation® technology and ExPERT™ platform to accelerate the development of its solid tumor cell-therapy candidates NEW YORK and GAITHERSBURG, Md., Jan. 31, 2022 (GLOBE NEWSWIRE) — MaxCyte, Inc., (Nasdaq: MXCT; LSE: MXCT, MXCN), a leading provider of enabling platform technologies for ex-vivo cell engineering, today announces the signing of a strategic platform license (SPL) with Intima Bioscience, Inc., a clinical stage biotechnology company developing genetically engineered cell therapies for solid tumor cancer. Intima joins a group of 15 other leading cell therapy companies who have partnered with MaxCyte.

Under the terms of the agreement, Intima obtains non-exclusive clinical and commercial rights to use MaxCyte’s Flow Electroporation® technology and ExPERT™ platform. In return, MaxCyte is entitled to receive platform licensing fees and program-related milestone payments.

Intima is currently running a Phase 1/2 clinical study (NCT04426669) of its lead checkpoint cell therapy candidate, which targets the immune checkpoint CISH in patients with gastrointestinal and colon cancers.

“As we advance our innovative eTIL CISH knock out programs, we are preparing for the future by securing access to MaxCyte’s industry leading cellular engineering technology,” said Alessandro Riva, M.D., Intima CEO.

Doug Doerfler, President and CEO of MaxCyte, said: “We are proud to be working with Intima, a cell therapy leader leveraging the unique power of genetically engineered tumor infiltrating lymphocytes (eTIL). This relationship represents an important achievement for MaxCyte as it expands the use of our next-generation technology platform to support engineering in additional novel cell types.”

MaxCyte’s ExPERT™ instrument portfolio is the next generation of leading, clinically-validated, electroporation technology for complex and scalable cell engineering. By delivering high transfection efficiency, seamless scalability and enhanced functionality, the ExPERT™ platform delivers the high-end performance essential to enabling the next wave of biological and cellular therapeutics. Intima Bioscience is MaxCyte’s 16th SPL, which generate pre-commercial milestone revenue and the vast majority of which include post-commercial revenue.

About MaxCyte

MaxCyte is a leading commercial cell-engineering company focused on providing enabling platform technologies to advance innovative cell-based research as well as next-generation cell therapeutic discovery, development and commercialization. Over the past 20 years, we have developed and commercialized our proprietary Flow Electroporation® platform, which facilitates complex engineering of a wide variety of cells. Our ExPERT™ platform, which is based on our Flow Electroporation technology, has been designed to support the rapidly expanding cell therapy market and can be utilized across the continuum of the high-growth cell therapy sector, from discovery and development through commercialization of next-generation, cell-based medicines. The ExPERT family of products includes: four instruments, the ATx™, STx™ GTx™and VLx™; a portfolio of proprietary related processing assemblies or disposables; and software protocols, all supported by a robust worldwide intellectual property portfolio.

About Intima Bioscience

Intima Bioscience is a company advancing checkpoint cell therapy, an approach that combines the use of patients’ own immune cells with immune checkpoint targeting, in solid tumors with curative intent. Intima is currently running a Phase 1/2 clinical study of its checkpoint cell therapy, which targets the immune checkpoint CISH in patients with gastrointestinal and colon cancers.

MaxCyte Contacts:

US IR Adviser Gilmartin Group David Deuchler, CFA

+1 415-937-5400  ir@maxcyte.com

Nominated Adviser and Joint Corporate Broker Panmure Gordon Emma Earl / Freddy Crossley Corporate Broking Rupert Dearden

+44 (0)20 7886 2500

UK IR Adviser Consilium Strategic Communications Mary-Jane Elliott Chris Welsh

+44 (0)203 709 5700 maxcyte@consilium-comms.com

ROCKVILLE, MD. (PRWEB)  FEBRUARY 02, 2022

Data from a Total of Five Patients Demonstrates Critical Markers of the Company’s HIV Cure Gene & Cell Therapy American Gene Technologies (AGT), a clinical-stage biotechnology company working to cure HIV, announced that it has reached another important milestone for its HIV cure program. Five participants were treated with AGT103-T and are stably engrafted with genetically modified cells.

Laboratory studies confirmed substantial increases in virus-specific T cells consistent with improved immunity against HIV in all participants. The early data addresses key trial endpoints and is aligned with the objective of restoring natural immunity against HIV.

“These latest laboratory studies of clinical material are extremely encouraging,” said AGT CEO Jeff Galvin. “To see these markers in all five trial participants indicates that prospects are bright for a potential one-and-done therapy to functionally cure HIV. This milestone brings us another step closer to our goal of returning people living with HIV to a normal life without the side effects of ART and having no further consequences of HIV.”

To date, all participants that have been infused with the cell and gene therapy product AGT103-T are engrafted with modified cells and are displaying enhanced antiviral T cell immunity. The treatment withdrawal studies are planned to begin in Q2 and will test the efficacy of this therapy. AGT is moving closer to the goal of allowing people living with HIV to discontinue daily medication without fear of developing AIDS or infecting others.

Phase 1 Trial Background

• Overview: Designated the RePAIR trial (Restore Potent Antiviral Immune Responses, NCT04561258), AGT’s first-in-human study for AGT103-T is currently underway at trial sites in the Maryland / Washington, D.C. area. Participants in the Phase 1 trial receive a single infusion of their own HIV-specific CD4 T cells after those cells are enriched and genetically modified to resist infection. Without any observed negative or adverse events, the DSMB has allowed the trial to continue without adjustments or delays.
• Focus: The primary endpoints of RePAIR are safety and efficacy. Testing related to secondary endpoints evaluates responses to treatment, including changes in the immune response to HIV.
• Participant Criteria: Participants range in age from 18 to 60 and include males and females. Participants have been diagnosed with HIV for at least three years and must have taken HIV antiretroviral medication for more than two years prior to enrollment. Participants cannot be pregnant and must be available to attend 17 study visits over a 10-month period. Anticipated completion of treatments in the Phase 1 study is Q3 2022, although final data and long-term monitoring will continue. The Phase 1 study will include up to 18 participants. (The recruitment status of the Phase 1 RePAIR clinical trial, along with information on the trial sites, can be found on the official ClinicalTrials.gov website.)
• Timeframe: Participants treated with AGT103-T are followed for 6 months in this safety study before enrolling in an FDA-mandated, 15-year, long-term follow up (required for all gene therapy trials). The first infusion occurred in May 2021, the second in August 2021, the third in September 2021, the fourth and fifth in November 2021, and the sixth in February 2022. A treatment withdrawal study to determine product efficacy is planned for Q2 of 2022.

About HIV

According to UNAIDS, approximately 37.7 million people worldwide live with HIV/AIDS. In the United States, government statistics show 1.2 million people have HIV and estimate that 34,800 Americans were newly infected with HIV in 2019. Across the globe, UNAIDS estimates that approximately 1.5 million individuals were newly infected with HIV in 2020. The Washington D.C./Baltimore area is often cited as a ‘hot spot’ for HIV, with Washington, D.C., having the highest rate of infection at nearly 46 cases per 100,000 population and Baltimore City having rates of 17 cases per 100,000. Maryland also ranks sixth among U.S. states and territories in HIV diagnosis rates, with more than 900 new cases in 2019 alone, according to the Maryland Department of Health.

Since the late 1980s, antiretroviral drugs have restored quality of life to persons living with HIV and, in some cases, have even been used to prevent new infections. However, no approved treatment has demonstrated the ability to cure HIV. AGT is committed to addressing this unmet medical need.

About AGT103-T

AGT103-T is a genetically modified cell product made from a person’s own cells. AGT’s unique approach focuses on permanently repairing the key immune system damage caused by HIV. AGT’s goal is to develop a cell and gene therapy capable of repairing the immune system so it will provide natural control over HIV replication.

About American Gene Technologies AGT is a gene and cell therapy company with a proprietary gene-delivery platform for rapid development of cell and gene therapies to cure infectious diseases, cancers, and inherited disorders. AGT’s mission is to transform people’s lives through genetic medicines that rid the body of disease. AGT has been granted four patents for the technology used to make AGT103-T and 11 patents for its unique immuno-oncology approach to stimulate gamma-delta (γδ) T cells to destroy a variety of solid tumors. The company has developed a synthetic gene for treating Phenylketonuria (PKU), a debilitating inherited disease. AGT’s treatment for PKU has been granted Orphan Drug Designation by the Food and Drug Administration (FDA), and it is expected to reach the clinic in 2022.

Video: https://youtu.be/fiA2s7JCkJ8

Media Contact: Kathy Fowler American Gene Technologies Phone: (410) 963-2345 Email: kathleen@onthemarcmedia.com

Johns Hopkins researchers Nickolas Papadopoulos, Ken Kinzler and Bert Vogelstein have spent their careers working on ways not just to treat cancer but to detect it before it becomes a threat. The goal: a blood test for the earlier detection of cancer incorporated into routine medical care. Their dream is closer to reality thanks in part to a $2.15 billion acquisition of their company, Thrive Earlier Detection Corp., one year ago by Exact Sciences Corp., a global leader in cancer-detection testing.

The heart of the researchers’ work is the liquid biopsy, a test done on a blood sample to look for signals derived from cancer cells circulating in the blood. In 2011, they invented SafeSeqS, a next-generation gene sequencing technology that simultaneously and individually analyzed millions of DNA molecules to identify mutations in the bloodstream more accurately than other methods.

Two years later, SafeSeqS was incorporated into a pilot study of their liquid biopsy test, which used cervical fluid obtained during routine Pap tests to detect endometrial and ovarian cancer. The “PapGene” test accurately detected all 24 endometrial cancers and nine of 22 ovarian cancers in the study. In 2018, the researchers reported that PapSEEK, the screening test, was nearly 99% specific for cancer and detected 81% of endometrial cancers and 33% of ovarian cancers in a study of 2,000 samples from nearly 1,700 women.

Click here to more via JHU.

The pandemic, now approaching year three in full force, has impacted nearly every facet of the life sciences’ job market. A talent shortage existed pre-pandemic and has only accelerated since then. For life sciences employers, finding and securing talent will continue to be a major challenge throughout 2022.

However, like most disruptive forces, the resulting turmoil and upheaval created new opportunities and the impetus, or one might say necessity, to innovate. Talent acquisition strategies and tactics haven’t escaped disruption; the manner by which employers find, hire, and onboard new employees has had to adapt and evolve during the pandemic.

Life sciences talent teams have risen to pandemic challenges to create an entirely new hiring process and ecosystem in short order. This new process seemed strange at first, even awkward and alien, but once the dust settled an innovative new approach spearheaded by a move to remote methods emerged, yielding greater efficiencies.

Much like commercial real estate and office space design will never be the same, the talent acquisition game has irrevocably changed. And some would say this change is for the better.

Let’s take a look back on some of these pandemic-inspired changes to talent acquisition that have taken root in response to the pandemic.

• Descartes-25 engineered to deliver a combination of synergistically active anti-myeloma therapies directly to tumor microenvironment
• Product generated from Company’s Part 1271-compliant Master Cell Bank of human umbilical cord Mesenchymal Stem Cells
• Fifth IND in the five years since Cartesian was founded

Gaithersburg, Md., January 25, 2022 – Cartesian Therapeutics, a fully integrated clinical-stage biotechnology company pioneering RNA cell therapy in and beyond oncology, today announced that it has dosed the first patient in a Phase 1/2a multicenter clinical study evaluating Descartes-25 in patients with multiple myeloma.  To the company’s knowledge, Descartes-25 is the first off-the-shelf RNA cell therapy to enter clinical trials for any cancer and marks the company’s fifth FDA Investigational New Drug (IND) allowance in five years.  Descartes-25 is produced at Cartesian’s wholly owned cGMP manufacturing facility with the company’s proprietary RNA Armory^® cell manufacturing platform.  This platform now includes an internally developed, Part 1271-compliant Master Cell Bank of human umbilical cord Mesenchymal Stem Cells (MSC) that was used to engineer Descartes-25.

Descartes-25 is designed to deliver two complementary antitumor proteins directly to the tumor:  a novel three-arm bispecific antibody that binds B-cell Maturation Antigen (BCMA) with femtomolar avidity and the potent antitumor cytokine interleukin-12 (IL-12).  Descartes-25 cells are further engineered with a membrane-bound homing protein that directs the cells to the tumor microenvironment for local delivery of their antitumor cargo.  In preclinical models, Descartes-25’s IL-12 synergistically potentiates its BCMA bispecific antibody to eliminate myeloma with unprecedented activity.

“Patients with relapsed and/or refractory multiple myeloma have few treatment options remaining,” said Kenneth Anderson, M.D., Kraft Family Professor of Medicine at Harvard Medical School and Director of the Jerome Lipper Multiple Myeloma Center and LeBow Institute for Myeloma Therapeutics.  “A cell therapy that locally delivers a combination of a BCMA-bispecific antibody and IL-12, without using lymphodepleting chemotherapy, is an elegant and highly innovative approach.  If approved, it will be a welcome addition to our toolkit for treating this currently incurable disease.”

“We designed Descartes-25 to be highly potent and well tolerated by focusing on creating the ideal pharmacokinetic profile: continuous, measured, and local delivery of a synergistic combination of antitumor agents,” said Murat Kalayoglu, M.D., Ph.D., President and Chief Executive Officer at Cartesian.  “I am proud of our integrated team of scientists and physicians for their hard work and rapid clinical translation of this first-in-class therapy.”

“With Descartes-25, Cartesian scientists used RNA Armory^® technology to convert stem cells into a targeted combination therapy,” said Chief Scientific Officer Michael Singer, M.D., Ph.D.  “Therefore Descartes-25 is not just a potent antitumor therapy.  It’s also a blueprint for future RNA cell therapies to deliver three or more rationally selected and spatially targeted combination therapeutics for a diverse array of diseases.”

About the Phase 1/2a Clinical Trial

The Phase 1/2a study (NCT05113342) is an open-label, multicenter, dose escalation trial for Descartes 25 in patients with relapsed and/or refractory multiple myeloma.  The study aims to enroll twenty patients who have previously failed two or more lines of treatment to determine the safety and preliminary efficacy of Descartes-25.  For more information visit https://www.cartesiantherapeutics.com/clinical-trials/#oncology.

About RNA Armory^®

The RNA Armory^® is Cartesian’s proprietary cell engineering platform that generates large-scale, potent RNA cell therapies with extended protein expression.  The RNA Armory^® is currently focused on RNA engineering two types of cells, T-cells and Mesenchymal Stem Cells (MSCs).  Our CAR T-cell programs harness the safety of RNA and autologous engineering to target autoimmune diseases and frontline cancer – without lymphodepletion.  Our off-the-shelf MSC programs leverage these cells’ clinical safety record and excellent capacity for protein secretion to deliver synergistic combinations of therapies.  For more information visit https://www.cartesiantherapeutics.com/rna-cell-therapy/#technology.

About Cartesian Therapeutics

Founded in 2016 and with three assets in clinical trials, Cartesian is the leader in RNA cell therapy.  With a mission to unleash the potential and reach of cell therapy with RNA engineering, Cartesian is pioneering RNA cell therapies in and beyond oncology with products in development for autoimmune, oncologic and respiratory disorders.  All investigational therapies are manufactured at Cartesian’s wholly owned, state-of-the-art cGMP manufacturing facility in Gaithersburg, MD, enabling complete control of product quality, production schedules and costs while accelerating clinical translation of discoveries. For more information visit www.cartesiantherapeutics.com.

Media Contacts:

Maggie Beller for Cartesian Therapeutics
Russo Partners, LLC
maggie.beller@russopartnersllc.com

646-942-5631

Center will expand Orgenesis’ Point of Care (POCare) Platform Capabilities in Maryland GERMANTOWN, Md., Jan. 18, 2022 (GLOBE NEWSWIRE) — Orgenesis Inc. (NASDAQ: ORGS) (“Orgenesis” or the “Company”), a global biotech company working to unlock the full potential of cell and gene therapies, and The Johns Hopkins University, today announce the next phase of their collaboration. This new phase involves construction of a cell and gene therapy processing facility for point of care treatment of patients at Johns Hopkins which is planned to start in Q2 2022 and is expected to be operational in Q2 of 2023.

Construction of the new POCare Center, also known as the Maryland Center for Cell Therapy Manufacturing, has been funded in part by a $5 million grant from the State of Maryland. The new state-of-the-art 7,000-square-foot facility has been designed to meet U.S. Food and Drug Administration standards and provides Johns Hopkins clinicians and researchers with a more streamlined path to treat patients and take promising and novel treatments from the lab to patient trials. This path will enable local capacity for processing of clinical therapeutics at the point of care, rather than having to outsource clinical trial cell and gene therapy manufacturing to third parties.

In addition, the establishment of the new POCare Center will enable rapid scale up of additional processing capacity through connecting/servicing Orgenesis Mobile Processing Units and Labs (OMPULs). OMPULs shorten the implementation time of new capacity from 18-24 months to 3-6 months. Each POCare Center can service multiple OMPULS. The first OMPUL in Maryland is expected to deploy 2H of 2022.

Orgenesis currently plans to base 30 of their own employees on the site when it is completed. Orgenesis is already using lab space at FastForward, Johns Hopkins Technology Ventures’ (JHTV) innovation hub, as the acting POCare Center for the Mid-Atlantic Region.

“I’d like to congratulate Orgenesis as they begin building the company’s new Maryland Center for Cell Therapy Manufacturing on the Johns Hopkins University campus in Baltimore. The state is pleased to support this project with a $5 million grant to assist with construction,” said Maryland Governor Larry Hogan.  “With the current demands on the healthcare industry, we are acutely aware of the need to continue to position Maryland in the forefront of biomedical advancements. This center will help develop innovative new therapies to improve the lives of patients for years to come.”

“Orgenesis continues to develop and extend key partnerships within its international POCare Network. These international partnerships are now experiencing significant investment and construction across the globe to build on the achievements within the Network, as illustrated by our expanded collaboration with Johns Hopkins,” said Vered Caplan, CEO, Orgenesis. “We are honored to work with Johns Hopkins, America’s first research university and home to nine world-class academic divisions working together in one university. The POC Center at Johns Hopkins will help propel the development of therapies targeting a range of conditions that directly affect the lives of millions of patients.”

About Orgenesis Orgenesis is a global biotech company working to unlock the full potential of cell and gene therapies (CGTs) in an affordable and accessible format at the point of care. The Orgenesis POCare Platform is comprised of three enabling components: a pipeline of licensed POCare Therapeutics that are processed and produced in closed, automated POCare Technology systems across a collaborative POCare Network. Orgenesis identifies promising new therapies and leverages its POCare Platform to provide a rapid, globally harmonized pathway for these therapies to reach and treat large numbers of patients at lowered costs through efficient, scalable, and decentralized production. The POCare Network brings together patients, doctors, industry partners, research institutes and hospitals worldwide to achieve harmonized, regulated clinical development and production of the therapies. www.orgenesis.com.

Notice Regarding Forward-Looking Statement This press release contains forward-looking statements which are made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities and Exchange Act of 1934, as amended. These forward-looking statements involve substantial uncertainties and risks and are based upon our current expectations, estimates and projections and reflect our beliefs and assumptions based upon information available to us at the date of this release. We caution readers that forward-looking statements are predictions based on our current expectations about future events. These forward-looking statements are not guarantees of future performance and are subject to risks, uncertainties and assumptions that are difficult to predict. Our actual results, performance or achievements could differ materially from those expressed or implied by the forward-looking statements as a result of a number of factors, including, but not limited to, our reliance on, and our ability to grow, our point-of-care cell therapy platform, our ability to effectively use the net proceeds from the sale of Masthercell, our ability to achieve and maintain overall profitability, the sufficiency of working capital to realize our business plans, the development of our POCare strategy, our trans differentiation technology as therapeutic treatment for diabetes which could, if successful, be a cure for Type 1 Diabetes, the technology behind our in-licensed ATMPs not functioning as expected, our ability to retain key employees, our competitors developing better or cheaper alternatives to our products and the risks and uncertainties discussed under the heading “RISK FACTORS” in Item 1A of our Annual Report on Form 10-K for the fiscal year ended December 31, 2019, and in our other filings with the Securities and Exchange Commission. We undertake no obligation to revise or update any forward-looking statement for any reason.

IR contact for Orgenesis: Crescendo Communications, LLC Tel: 212-671-1021 Orgs@crescendo-ir.com

Media contact for Orgenesis: Image Box Communications Neil Hunter / Michelle Boxall Tel +44 (0)20 8943 4685