Interius BioTherapeutics, Inc.

The first shards of human data on in vivo CAR-T cell therapy look promising, based on recent separate reports from two biotech companies’ academic-led studies in China. Last week, a Chinese startup called Genocury became the second company to share initial clinical data on a CAR-T treatment that doesn’t require a patient’s cells to be extracted from their body. Genocury said that an advanced large B cell lymphoma patient went into complete remission after one month of in vivo CAR-T therapy, and has remained in remission for over three months. Notably, the in vivo CAR-T therapy does not require lymphodepletion, a course of chemotherapy that conventional CAR-T therapies require before treatment and that comes with toxicities. “This is good news for the field and demonstrates the increasing momentum around in vivo CAR-T approaches,” said University of Pennsylvania CAR-T pioneer Bruce Levine, who isn’t involved in Genocury’s studies but is a scientific founder of another in vivo CAR-T company called Capstan. Last week’s results follow Belgian startup EsoBiotec’s announcement in January that a multiple myeloma patient who received the lowest dose of its in vivo CAR-T therapy had no detectable cancer cells in their bone marrow at one month, without any significant adverse events during treatment. That patient also did not receive prior lymphodepletion. The data from both companies come from investigator-initiated studies in China, which biotech startups have been leveraging more frequently to quickly generate early clinical data on their experimental treatments. EsoBiotec is working with Pregene Biopharma which, like Genocury, is based in Shenzhen. In March, AstraZeneca announced plans to acquire EsoBiotec for $425 million, plus additional payments. At least five groups have begun clinical trials for in vivo CAR cell therapies. Aside from EsoBiotec and Genocury, Myeloid Therapeutics, Interius BioTherapeutics and Umoja Biopharma are all studying experimental in vivo cell therapies in patients. In conventional CAR-T therapy, a patient’s cells are extracted, engineered, and then reinfused — a complex, expensive process that can take weeks to months. The therapy, while transformative for certain blood cancers, comes with safety risks from the lymphodepletion before treatment and immune overreaction (cytokine release syndrome and ICANS) afterwards. And so far, comparatively less complex “off-the-shelf” treatments using donor cells have yet to take off. Makers of in vivo cell therapy hope that their treatments can become a different kind of “off-the-shelf” option. Their therapies deliver genetic material to turn the patient’s immune cells into cancer-fighting ones directly in the body. The companies are using different technologies to deliver their treatments and are also going after a range of diseases. The delivery technologies fall into two camps: viral and non-viral. Researchers have more concerns around the potential risks of a virus-based therapy, including of secondary cancer, but it’s an older and more proven approach. Non-viral delivery is newer and more challenging, and fewer companies have reached clinical studies. It is unclear based on Genocury’s disclosures whether it is using a viral or non-viral approach. A photo on its website mentions both viral and non-viral methods, and the company could not be reached for comment. EsoBiotec is developing a lentiviral-based cell therapy that goes after BCMA, a well-known target for multiple myeloma. Interius, which expects to report clinical data later this year, and Umoja are likewise studying lentiviral-based therapies. Umoja, like Genocury, is developing a CD19-targeted CAR-T therapy for blood cancer, and it plans to study the treatment in autoimmune disease as well. AbbVie has an option to license that therapy, and in January the startup raised $100 million to jumpstart its clinical studies. Interius’ therapy targets another immune cell protein called CD20, and it’s studying its B cell cancer therapy in both Australia and Germany. The Philadelphia biotech’s therapy is meant to make both ​​CAR-T and NK cells directly in the body. On the other hand, Sid Mukherjee’s startup Myeloid is using mRNA delivered non-virally. It uses fatty acid envelopes called lipid nanoparticles — similar to what’s used in the Covid vaccines. However, as its name suggests, it’s targeting a type of immune cell called myeloid cells. It has two programs in the clinic going after solid tumors — an area of cancer that conventional CAR-T therapies have yet to crack.

PHILADELPHIA, April 17, 2025 (GLOBE NEWSWIRE) -- Interius BioTherapeutics, a clinical-stage company engineering targeted, programmable vectors for the precision delivery of genetic medicines, today announced that it will present at several upcoming scientific meetings. Cellicon Valley '25: The Future of Cell and Gene TherapiesPhiladelphia, PAApril 30 – May 2, 2025 Plenary Session: Are In Vivo Cell Therapies the Key to the Future of Medicine?Presentation Title: Cell-specific in vivo gene delivery: A pipeline dream no longerPresenter: Philip Johnson, M.D., Chief Executive Officer, Interius BioTherapeuticsPresentation Date and Time: Thursday, May 1, at 3:50 pm EDT International Society for Cell and Gene Therapy (ISCT) 2025 Annual MeetingNew Orleans, LAMay 7 – 10, 2025 Scientific Session: In Vivo Cell Engineering: Breaking Through the Manufacturing BottleneckPresentation Title: INVISE: A first-in-human Phase 1 clinical trial evaluating the safety of INT2104 for in vivo generation of CAR T and CAR NK cells in adults with relapsed/refractory B cell malignanciesPresenter: Dr. David Bishop, Haematologist, Westmead Hospital, Sydney, AustraliaPresentation Date and Time: Friday, May 9, at 9:15 am CT American Society of Gene and Cell Therapy (ASGCT) 28th Annual MeetingNew Orleans, LAMay 13 – 17, 2025 Scientific Symposium: The Coalition of International Gene Therapy Societies Showcases: Moving from ex vivo Cell Therapies to in vivoPresentation Title: Investigational in vivo CAR-T therapy designed to treat B-cell malignanciesPresenter: Philip Johnson, M.D., Chief Executive Officer, Interius BioTherapeuticsPresentation Date and Time: Thursday, May 15, from 3:45 – 5:30 pm CTLocation: Room 265-268 About Interius BioTherapeutics Interius BioTherapeutics is a clinical stage biotechnology company engineering targeted, programmable vectors for the precision delivery of genetic medicines to treat an array of challenging diseases in oncology, autoimmunity and beyond. Leveraging a proprietary platform, Interius has created a differentiated off-the-shelf therapeutic modality designed for broad patient access. The Company’s lead programs aim to overcome the current limitations of ex vivo chimeric antigen receptor (CAR) T-cell therapy by creating therapeutic CAR cells directly in the patient’s body, avoiding preconditioning chemotherapy, manufacturing bottlenecks, and high costs.For more information, visit www.interiusbio.com and follow us on LinkedIn. Media ContactMichael RubensteinLifeSci Communicationsmrubenstein@lifescicomms.com +1 646-386-1613

AstraZeneca is bolstering its cell therapy capabilities, agreeing on Monday to pay up to $1 billion for a biotechnology startup making treatments that reprogram cells inside the body.The British drugmaker is buying EsoBiotech, a Belgian startup, for $425 million in cash upfront. EsoBiotechs investors, among them Invivo Partners and UCB Ventures, could receive up to $575 million in additional payouts should the startups programs hit certain development and regulatory milestones, AstraZeneca said in a statement.The deal hands AstraZeneca a technology designed to make cell therapies that can be administered through a single infusion, rather than via a complicated process that involves genetically manipulating cells in a lab. These so-called in vivo cell therapies, if successful, would help broaden the reach of traditional cell therapies, and be more convenient and accessible as well.Several biotechs are pursuing in vivo cell therapy research, among them well-funded startups like Capstan Therapeutics and Umoja Biopharma. Some, such as Interius BioTherapeutics and EsoBiotech, have programs in early clinical testing. EsoBiotech in December started human testing of a multiple myeloma cell therapy called ESO-T01.That therapy, like others EsoBiotech is developing, uses what it calls novel, highly targeted lentiviruses to deliver genetic instructions into immune cells. This reprogramming process is done inside the body through an intravenous injection that takes minutes rather than the multiday or week turnaround typically associated with CAR-T therapy, AstraZeneca said.AstraZeneca intends to use the technology to advance therapies for cancer as well as autoimmune diseases, the latter of which is a growing research focus for cell therapy companies. EsoBiotech has already been doing so with ESO-T01, which turns immune cells against a protein on B cells that become cancerous in multiple myeloma and malfunction in inflammatory disorders.EsoBiotechs technology has the potential to transform cell therapy and will enable us to scale these innovative treatments so that many more patients around the world can access them, said Susan Galbraith, the companys executive vice president of oncology and hematology research and development, in a statement.The acquisition is also the latest investment in cell therapy research by AstraZeneca. The company wasnt part of the initial wave of industry interest into this field. But over the last few years it has been building up internal capabilities and cutting deals with biotechs focused on newer approaches, such as donor-derived treatments or therapies made from different immune cells. An acquisition of Gracell Biotechnologies gave it an autoimmune cell therapy, too, though much of that research remains early.EsoBiotech will accelerate and expand the impact of our recent investments and marks a major step forward in realizing our ambition to harness the full potential of cell therapy, Galbraith said in the statement.More broadly, AstraZenecas interest could help rejuvenate a field thats been struggling through an investment slump. Large pharmaceutical companies desire for cell and gene therapy acquisitions could continue to grow as they look for ways to mitigate the impact of patent expirations and drug-price negotiations, wrote Baird analyst Jack Allen, in a Monday note to clients.[M]any speculate that large pharmas will need to execute acquisitions in order to preserve their top lines, he wrote, and cell and gene therapies could be appealing targets because of complicated manufacturing processes that could protect them from generic competition.While weve seen some M&A in this space to date, we believe an increase in large pharma/biotech appetite here could provide a key tailwind of this subsector, Allen added. '