Category Archives: Cell Therapy

AGOURA HILLS, Calif.--(BUSINESS WIRE)--A2 Biotherapeutics, Inc. (A2 Bio) is a biotechnology company focused on the development of a first-in-class, logic-gated Tmod CAR T cell therapy platform to tackle the fundamental challenge in solid tumor treatmentthe ability of cancer medicines to distinguish between tumor and normal cells. The Tmod system exploits irreversible genetic changes in cancer cells called loss of heterozygosity (LOH). A2 Bio will be presenting on Saturday, January 22nd at the American Society of Clinical Oncology (ASCO) Gastrointestinal (GI) Cancers Symposium taking place virtually and in-person on January 2022, 2022, at the Moscone Center in San Francisco, CA.

A2 Bio in partnership with Tempus, a leader in artificial intelligence and precision medicine, analyzed the Tempus xT NGS assay database that has over 21,000 samples with HLA locus LOH data and over 10,000 of which are from patients with advanced disease stage 3; 3,000 of these samples are from GI cancer patients. These results demonstrate clonal frequency of HLA LOH in advanced GI solid tumor cancers of 16.3%, with a range of 15.6%-20.8% among colorectal, pancreatic, and gastroesophageal tumors. In addition, these frequencies are similar to primary tumors from The Cancer Genome Atlas (TCGA) database. Thus, HLA LOH is a well-defined discriminator between tumor and normal cells and can be exploited for NOT logic-gated Tmod CAR T to reduce on-target off-tumor toxicity. These results expand knowledge about HLA LOH in these tumors and pave the way for the use of Tempus xT NGS in screening patients for future Tmod CAR T cell therapy.

A2 Bio has developed Tmod CAR T, which targets clonal HLA LOH as a clear differentiator between normal versus tumor cells, said William Go, MD, PhD, Chief Medical Officer at A2 Bio. The Tempus xT next generation sequencing assay will be utilized in BASECAMP-1. BASECAMP-1 is a study to identify HLA LOH solid tumor cancer patients and obtain their T cells earlier in their treatment paradigm who may benefit from future carcinoembryonic antigen (CEA) and mesothelin (MSLN) Tmod CAR T therapy. Novel therapies are sorely needed in GI malignancies, especially in pancreatic cancer, where limited therapeutic advances have been made in over two decades.

Lead author J. Randolph Hecht, MD, Professor of Clinical Medicine, Director of the UCLA Gastrointestinal Oncology Program and principal investigator of the BASECAMP-1 trial, stated: Tmod CAR T is an elegant and truly novel therapeutic utilizing a NOT logic gate to distinguish between normal and tumor cells. While other T cell therapies have failed in GI cancers due to toxicity or lack of efficacy, targeting HLA LOH with Tmod may provide the therapeutic safety window necessary for clinically significant outcomes. In addition, patients who might benefit from this approach can be identified using the widely used Tempus xT NGS assay early in the course of their disease.

ASCO GI Poster Information

Title: Next generation sequencing (NGS) to identify relapsed gastrointestinal (GI) solid tumor patients with human leukocyte antigen (HLA) loss of heterozygosity (LOH) for future logic-gated CAR T therapy to reduce on target off tumor toxicity

Presenter: J. Randolph Hecht, MD, Director of the UCLA Gastrointestinal Oncology Program

Session: Poster Session C: Cancers of the Colon, Rectum, and Anus.

Date/Time: Saturday, January 22 at 6:30 am to 7:55 am and 12:30 pm to 2:00 pm PT

Location: Abstract 190 - Level 1, West Hall; Moscone Center, San Francisco, CA

A2 Bios posters presented can be viewed on the companys website at http://www.a2bio.com/science/abstracts-and-publications.

About A2 Biotherapeutics

A2 Biotherapeutics has invented the Tmod cell therapy platform to tackle the fundamental challenge in solid tumor treatmentthe ability of cancer medicines to distinguish between tumor and normal cells. The Tmod mechanism utilizes two receptors to exploit common, specific gene losses in tumors which demarcate the tumor from normal cells. A2 is positioned to deliver a broad pipeline of both autologous and allogeneic cell products, with in-house cGMP manufacturing, a leadership team with 90+ years combined experience in biotech and cell therapy, and a world-class scientific advisory board. A2 is backed by investors that include The Column Group, Vida Ventures, Samsara BioCapital, Nextech Invest, Casdin Capital, Euclidean Capital, UC Investments (Office of the Chief Investment Officer of the Regents), Hartford HealthCare Endowment, StepStone Group, Schroders, Section 32 and Merck.

For more information, please visit http://www.a2bio.com and linkedin.com/a2-biotherapeutics.

Excerpt from:
A2 Bio to Present at ASCO-GI 2022 on Use of Next Generation Sequencing (NGS) to Identify GI Cancer Patients for Future Tmod CAR T Therapy - Business...

REGULATED INFORMATION

Recruitment for ALLOB tibial facture Phase IIb study ongoing and on schedule to release topline data in Q1 2023, despite COVID slowdown

New scientific advisory board appointments to bolster the further development of the next generation iMSCg platform

Discussions for ALLOB global partnership still ongoing. Completion of potential global partnership delayed and now anticipated in Q1 2022

Financial position strengthened following equity fundraising in Q4 2021 with runway expected into Q3 2022

Gosselies, Belgium, 19 January 2022, 7am CET BONE THERAPEUTICS (Euronext Brussels and Paris: BOTHE), the cell therapy company addressing unmet medical needs in orthopedics and other diseases, announces today a business update for the fourth quarter, ending 31 December 2021 as well as a business outlook for 2022.

Bone Therapeutics activity in Q4 2021 has resulted in a broadened pipeline and stronger therapeutic potential for the company. The pandemic continues to be a concern across the cell and gene therapy, biopharma and healthcare industries. Bone Therapeutics has, however, put measures in place to reduce the impact of the pandemic on its clinical development., said Miguel Forte, Chief Executive Officer, Bone Therapeutics. In addition, the deal for the global rights for the allogeneic osteoblastic cell therapy product ALLOB, when and if completed, will allow Bone Therapeutics, together with its partners, to ensure the development of ALLOB towards commercialization, while at the same time continue to explore options to expand the iMSCg platform. This includes the development of a next generation of genetically engineered mesenchymal stromal cells and the use of highly scalable and versatile cell sources such as induced pluripotent stem cells. Bone Therapeutics has expanded its Scientific Advisory Board purely for this purpose.

Operational highlights

Bone Therapeutics allogeneic bone cell therapy product, ALLOB is currently being evaluated in a randomized, double-blind, placebo-controlled Phase IIb study in patients with high-risk tibial fractures. Bone Therapeutics anticipates finalizing patient recruitment of this study in 2022. This finalization is subject, as across the industry, to evolution of the ongoing COVID-19 pandemic and the associated containment measures. Although early recruitment rates were very promising, the recruitment rates have temporarily slowed in recent months due to pandemic-related factors, such as reduced site activities due to staff availability and the number of available patients due to less occurrence of accidents. Bone Therapeutics has already implemented several mitigating measures in collaboration with the involved clinical research organization to improve and facilitate recruitment. These measures include site expansion, training, information, best practices sharing and close monitoring of progress. As a result of these measures Bone Therapeutics continues to currently expect the release of topline data by Q1 2023.

In November 2021, Bone Therapeutics signed a non-binding term sheet for the global rights for ALLOB, Bone Therapeutics allogeneic osteoblastic cell therapy product, with one of its current Chinese partners, Link Health Pharma Co., Ltd (Link Health). If the licensing deal is concluded, the partner would be responsible for all future costs of development of ALLOB, including the ongoing ALLOB TF2 Phase IIb trial and costs related to development, process development (scale up) and manufacturing of the product. In this non-binding agreement, no upfront or development milestones are expected but Bone Therapeutics would receive commercial milestone payments of up to 60 million in total and tiered royalties on net sales of up to 25%. The negotiations for the global rights agreement are still ongoing but take longer than expected. The envisaged completion of a final binding agreement has been delayed and is now contemplated over the course of Q1 2022.

Story continues

Corporate highlights

In October 2021, Bone Therapeutics appointed key experts to its Scientific Advisory Board (SAB). The members of the SAB consist of world-recognized scientists and clinicians in the cell and gene therapy field. The additional members of the SAB have been added to provide additional expert guidance on the development of Bone Therapeutics novel, next generation induced pluripotent stem cell-derived mesenchymal stromal cell (iMSCg) platform.

Bone Therapeutics has relocated its corporate offices to the Louvain-la-Neuve Science Park in Mont-Saint-Guibert (Louvain-la-Neuve), Belgium. Louvain-la-Neuve is home to the Catholic University of Louvain (UCLouvain), one of Belgiums premier academic research institutes. Bone Therapeutics will be part of a vibrant biotech ecosystem with a high concentration of cell therapeutic companies.

Financial highlights (1)

In December 2021, Bone Therapeutics raised additional 3.3 million funding through a private placement with current and new institutional investors to advance its lead orthopedic asset, ALLOB, through mid-stage clinical development. The funds will also support the development of the new iMSCg cell and gene therapy platform to address a broader array of underserved clinical indications outside orthopedics.

Disciplined cost and cash management will remain a key priority. Net cash burn for the full year 2021 is anticipated to be 16-18 million (1). The net cash position totaled 9.5 million (1) for the year ended December 31, 2021. Taking into consideration the 3.3M in gross proceeds from the private placement in the fourth quarter of 2021 and anticipated milestone payments, Bone Therapeutics expects to have sufficient cash to carry out its business objectives into Q3 2022. This assumes normal operation, as there may be further effects of the ongoing COVID-19 pandemic.

Outlook for 2022

Bone Therapeutics will continue to explore options to develop its allogeneic differentiated MSC based cell therapy platform, beyond ALLOB, into other therapeutic indications. If financing so allows, Bone Therapeutics is also aiming to expand its preclinical and clinical pipeline to additional indications by enhancing and professionalizing the therapeutic capacity of its cell and gene therapy platform. This activity includes the development of a next generation of genetically engineered mesenchymal stromal cells (MSCs) and the use of highly scalable and versatile cell sources such as induced pluripotent stem cells (iPSC).

For the ongoing Phase IIb ALLOB clinical study in difficult tibial fractures, Bone Therapeutics clinical team, in partnership with its clinical research organization, is continuing to institute measures to mitigate the impact of the pandemic and will closely monitor the recruitment progress. As a result of the initial mitigation actions, Bone Therapeutics continues to expect to report topline results as scheduled by the first quarter of 2023. However, a delay cannot be excluded. Should the pandemic continue to have impact on patient availability, Bone Therapeutics may have to re-evaluate this timeline and, in that eventuality, will communicate again to the market.

Bone Therapeutics will continue to prepare its discussions with the US FDA (Food and Drug Administration) in anticipation of the next steps in the clinical development of ALLOB in the US.

Bone Therapeutics is in the process of considering mandating a third party organization to explore partnership, M&A opportunities and alternative sources of funding for the iMSCg platform.

Link Health and Pregene, Bone Therapeutics partners in Asia, continue to drive the development of ALLOB towards the approval of the Investigational New Drug Application (IND) with the Chinese National Medical Products Administration (NMPA).

Bone Therapeutics continues to hold ALLOB business discussions with partners and aims to complete and to fully execute an agreement for the global rights for ALLOB over the course of Q1 2022.

Financial Calendar 2022

27 April Full Year Results & Annual Report 2021

25 May Q1 2022 Business and Financial Highlights

8 June Annual General Meeting 2022

7 September Half Year Results 2022

25 October Q3 2022 Business and Financial Highlights

The financial calendar is communicated on an indicative basis and may be subject to change.

(1) Unaudited number

About Bone Therapeutics

Bone Therapeutics is a leading biotech company focused on the development of innovative products to address high unmet needs in orthopedics and other diseases. The Company has a diversified portfolio of cell therapies at different stages ranging from pre-clinical programs in immunomodulation to mid stage clinical development for orthopedic conditions, targeting markets with large unmet medical needs and limited innovation.

Bone Therapeutics core technology is based on its cutting-edge allogeneic cell and gene therapy platform with differentiated bone marrow sourced Mesenchymal Stromal Cells (MSCs) which can be stored at the point of use in the hospital. Currently in pre-clinical development, BT-20, the most recent product candidate from this technology, targets inflammatory conditions, while the leading investigational medicinal product, ALLOB, represents a unique, proprietary approach to bone regeneration, which turns undifferentiated stromal cells from healthy donors into bone-forming cells. These cells are produced via the Bone Therapeutics scalable manufacturing process. Following the CTA approval by regulatory authorities in Europe, the Company has initiated patient recruitment for the Phase IIb clinical trial with ALLOB in patients with difficult tibial fractures, using its optimized production process. ALLOB continues to be evaluated for other orthopedic indications including spinal fusion, osteotomy, maxillofacial and dental.

Bone Therapeutics cell therapy products are manufactured to the highest GMP (Good Manufacturing Practices) standards and are protected by a broad IP (Intellectual Property) portfolio covering ten patent families as well as knowhow. The Company is based in the BioPark in Gosselies, Belgium. Further information is available at http://www.bonetherapeutics.com.

For further information, please contact:

Bone Therapeutics SAMiguel Forte, MD, PhD, Chief Executive OfficerLieve Creten, Chief Financial Officer ad interimTel: +32 (0)71 12 10 00investorrelations@bonetherapeutics.com

For Belgian Media and Investor Enquiries:BepublicBert BouserieTel: +32 (0)488 40 44 77bert.bouserie@bepublicgroup.be

International Media Enquiries:Image Box CommunicationsNeil Hunter / Michelle BoxallTel: +44 (0)20 8943 4685neil.hunter@ibcomms.agency / michelle@ibcomms.agency

For French Media and Investor Enquiries:NewCap Investor Relations & Financial CommunicationsPierre Laurent, Louis-Victor Delouvrier and Arthur RouillTel: +33 (0)1 44 71 94 94bone@newcap.eu

Certain statements, beliefs and opinions in this press release are forward-looking, which reflect the Company or, as appropriate, the Company directors current expectations and projections about future events. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial effects of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, competition and technology, can cause actual events, performance or results to differ significantly from any anticipated development. Forward looking statements contained in this press release regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, the Company expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this press release as a result of any change in expectations or any change in events, conditions, assumptions or circumstances on which these forward-looking statements are based. Neither the Company nor its advisers or representatives nor any of its subsidiary undertakings or any such persons officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in this press release or the actual occurrence of the forecasted developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release.

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Bone Therapeutics provides fourth quarter 2021 business update and 2022 outlook - Yahoo Finance

CancerNetwork, sat down with Yael Cohen, MD, to discuss the research that lead up to the phase 2 CARTITUDE-2 trial, examining the use of ciltacabtagene autoleucel for those with lenalidomiderefractory multiple myeloma

In an interview with CancerNetwork, Yael Cohen, MD, a senior physician in the Department of Hematology at Tel-Aviv Sourasky Medical Center, lent context to the updated findings of the phase 2 CARTITUDE-2 trial (NCT04133636), which were presented at the 2021 American Society for Hematology Annual Meeting & Exposition, examining ciltacabtagene autoleucel (cilta-cel) in patients with lenalidomide (Revlimid)refractory multiple myeloma.1

The CAR T-cell therapy was assessed in the phase 1b/2 CARTITUDE-1 trial (NCT03548207), which highlighted an overall response rate of 97.9% with a stringent complete response rate of 80.4% after a median follow-up of 18 months.2 The duration of these responses were examined in CARTITUDE-2, the results of which very encouraging, according to Cohen.

Transcript:

CARTITUDE-1 was testing cilta-cel in a very heavily pretreated group of patients; these were patients with relapsed/refractory myeloma after a median of 6 lines of therapy. These patients were known to have an expected very poor prognosis with any other alternatives available. Their overall response rate in CARTITUDE-1 was close to 98%. [Therefore], this was really a game changer. Actually [at] this [year's] ASH, there [were] the updated and very exciting results also on the durability of [these responses]. At 2 years, over 60% of the patients are still maintaining a response and the PFS was not reached.

Because they offer these attractive, exciting resultsas [with] other drugs in myelomathis is [being] taken to earlier lines of treatment; this is the CARTITUDE-2 trial. TheThis trial actually has several different cohorts and [in] cohort A, which is the study I'm presenting this evening, some patients [had] 1 to 3 lines of [prior] therapy. These are earlier patients [with] 20 patients [included]. These patients were on and then refractory to an [immunomodulatory agent] and proteosome inhibitors. [Additionally], 95% also had an alkylating agent [and] 65% had daratumumab [Darzalex]. These patients were also lenalidomide [Revlimid] refractory, so these are tough patients to get durable responses [in], as well. This was the population, and they got a single infusion of cilta-cel in this study.

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Yael Cohen, MD, on Background for and Key Findings from the Updated CARTITUDE-2 Trial With Cilta-Cel in Refractory Myeloma - Cancer Network

DUBLIN, Jan. 17, 2022 /PRNewswire/ -- The "Targeted Immunotherapy in Oncology" report has been added to ResearchAndMarkets.com's offering.

This research service investigates technologies that propel the development of new targeted immunotherapies, particularly those that deliver therapeutic DNA and proteins.

The top 3 growth opportunities focus on enhanced gene delivery systems for greater precision, the support of such systems through novel biotech platforms, and the affordability of precision medicine/targeted immunotherapies through modular, decentralized manufacturing systems.

Regarding technology, nonviral gene delivery and nucleic acid delivery offer significant advantages in terms of safety, particularly from immunogenicity and carcinogenicity. Not using viral vectors also makes the regulatory pathway and the biomanufacturing process more affordable because no additional precautions are needed; this, in fact, makes the price tag lower.

The study touches on the advantages and limitations of viral and nonviral gene delivery systems for targeted immunotherapies. Safety of administration without immunogenicity remains as the most relevant advantage. Liposomes have no replication risk and are less immunogenic than viruses.

The plurality of gene delivery systems is broadly benchmarked in terms of transfection efficiency, precision in cancer immunotherapy, and capability to pass the membrane barrier. As for remarkable innovation in biotech platforms, the research highlights gene addition as a novel approach that uses a delivery system to insert new genes directly into cells. The addition of a functional gene can take place either outside (ex vivo) or inside (in vivo) the body and can be used in cancer immunotherapy through CAR T-cell technology.

Gene delivery strategies are based on a comprehensive suite of clinically validated technologies, including electroporation, liposomes, nanoparticles, and nonviral and viral delivery modalities. Electroporation is best suited for efficient delivery to blood cells and immune cells ex vivo. Lipid nanoparticles (LNP) are better indicated for in vivo delivery to the liver and potentially other organs. Adenoassociated vectors are typically used for in vivo delivery to the eye and central nervous system (CNS).

As for optimal manufacturing process, closed-ended DNA (ceDNA) vectors exhibit a linear and continuous structure, which can be used for insertion of a transgene into a gene safe harbor (GSH) in the genome. For gene delivery, cell-targeted lipid nanoparticle (ctLNP) designed to avoid activation of the immune system upon initial dose can be used, while capsid-free approaches can be utilized to facilitate manufacturing.

This research profiles a remarkable number of companies succeeding in the targeted immunotherapy space. A competitive analysis of the top three innovators, based on their IP activity and patent innovation focus, is included. Funding and investment, including leading deals and investors focused on the technologies empowering targeted immunotherapies, are also covered.

Key Points Discussed:

1. Top innovations in biotechnology platforms focused on targeted immunotherapies2. Emerging technologies in gene and nucleic acids direct delivery systems3. Outstanding companies succeeding the targeted immunotherapy space4. Industry landscape and patent filing trends in the industry5. Major deals and investors focused on developing novel technologies for targeted immunotherapies6. Growth opportunities in the targeted immunotherapy area7. Best practices for risk management during the development of new technologies that enhance targeted immunotherapies

Key Topics Covered:

1.0 Strategic Imperatives1.1 Why Is It Increasingly Difficult to Grow? The Strategic Imperative: Factors Creating Pressure on Growth1.2 The Strategic Imperative1.3 The Impact of the Top Three Strategic Imperatives on Targeted Immunotherapies1.4 About The Growth Pipeline Engine1.5 Growth Opportunities Fuel the Growth Pipeline Engine1.6 Research Methodology

2.0 Growth Opportunity Analysis2.1 Rising Success of Gene and Cell Therapies, Gene Editing Techniques, and Novel Gene Delivery Platforms2.2 Framework for Personalized Targeted Immunotherapy2.3 Groundbreaking Immunotherapy Technology Platforms2.4 Fit-for-Purpose Immunotherapy Approaches2.5 Regulatory Environment2.6 Research Context2.7 Research Scope

3.0 The Need for Targeted Immunotherapy in Oncology3.1 Biotech Platforms for Cancer Immunotherapy3.2 Drivers and Accelerators3.3 Challenges and Restraints

4.0 Technology Assessment of Targeted Immunotherapies in Oncology4.1 Classification of Targeted Therapies4.2 Technologies Empowering Targeted Immunotherapies4.3 Germline Gene Editing Tools and Stem Cell Reprogramming4.4 Next-Generation Targeted Immunotherapy Requirements4.5 Viral and Nonviral Vector Delivery in Gene and Cell Therapies4.6 Process Opportunity Plays for Biopolymers & Bioprocessing in Gene Therapy4.7 Innovation Ecosystem and Collaboration Hubs4.8 Key Partnerships and Alliances in Gene Delivery4.9 Companies Focused on Viral and Nonviral Gene Delivery Systems in Targeted Immunotherapy4.10 Novel Synergies in Gene Delivery Systems Driving Targeted Immunotherapy Success4.11 Companies Focused on Gene Editing and Stem Cell Reprogramming Technologies4.12 Companies Focused on Medicinal Chemistry and Platform Chemistry Technologies

5.0 Pipeline Analysis of Targeted Immunotherapies in Oncology5.1 Principal Technologies Propelling Biotech Company Pipelines and Programs5.2 Clinical Validation Status of Nonviral Gene Delivery Systems5.3 Competitive Landscape5.4 State-of-the-Art Therapeutics Pipeline Dashboard: Inhibrx, Inc.5.5 State-of-the-Art Therapeutics Pipeline Dashboard: BioNTech SE5.6 State-of-the-Art Therapeutics Pipeline Dashboard: Adaptimmune Therapeutics llc.5.7 State-of-the-Art Therapeutics Pipeline Dashboard: Asher Bio5.8 State-of-the-Art Therapeutics Pipeline Dashboard: Gritstone bio5.9 State-of-the-Art Therapeutics Pipeline Dashboard: Jounce Therapeutics, Inc.

6.0 Stakeholders Leveraging Genome Tools for R&D6.1 In vivo Gene Transfer, Homology Medicines, US6.2 Next-Generation AAV Capsids, Abeona Therapeutics, US6.3 CRISPR Gene Editing, Editas Medicine, US6.4 AAV, Zinc Finger Nucleases, Sangamo Therapeutics, US6.5 Electroporation, Nonviral (LNP), Viral (AAV), Beam Therapeutics, US6.6 Customized AAV Vectors, 4D Molecular Therapeutics, US6.7 T-Cell Immunotherapy - Microfluidic Vortex Shedding, Indee Labs, US6.8 Precise Gene Editing Technology, Poseida Therapeutics, US6.9 Lentiviral Gene Delivery, Avrobio, US

7.0 Companies to Action7.1 Gene Therapy for Rare Diseases, Ultragenyx Pharmaceutical, US7.2 AAV Gene Delivery Technology, Spark Therapeutics (Roche), US7.3 Gene Replacement and Gene Knockdown, Voyager Therapeutics, US7.4 AAV Gene Delivery Technology, AGTC, US7.5 LV Gene Delivery and CAR T-cell Technology, BlueBird Bio, US7.6 DNA Nanoparticles, Copernicus Therapeutics, US

8.0 Funding and Investment Analysis8.1 Funding and Investment Assessment in Targeted Immunotherapy for Oncology8.2 Leading R&D Investments in Targeted Immunotherapy Platforms8.3 Principal Deals in Targeted Immunotherapy Platforms8.4 Leading Investors in Targeted Immunotherapy Platforms8.5 Investor Movements in Targeted Immunotherapy Platforms8.6 Government Funding in Targeted Immunotherapy Platforms8.7 Prime Contract Transactions in Targeted Immunotherapy Platforms8.8 Assistance Prime Transactions in Targeted Immunotherapy Platforms8.9 Venture Capital Funding Assessment in Targeted Immunogene Therapy8.10 Venture Capital Funding Deals in Targeted Immunogene Therapy

9.0 Patent Portfolio Analysis of Key Stakeholders9.1 Competitive Intelligence, Beam Therapeutics9.2 Competitive Intelligence, BlueBird Bio9.3 Competitive Intelligence, Generation Bio

10.0 Growth Opportunity Universe10.1 Growth Opportunity 1: Gene Delivery and Nucleic Acid Delivery for Rare and Genetic Diseases10.2 Growth Opportunity 2: Innovative Biotech Platforms for Precision Immunotherapy10.3 Growth Opportunity 3: Modular, Decentralized Manufacturing for Affordable Targeted Gene Immunotherapy10.4 Best Practices for Risk Management in Targeted Immunotherapy

11.0 Next Steps

For more information about this report visit https://www.researchandmarkets.com/r/fv6jsy

Media Contact:

Research and Markets Laura Wood, Senior Manager [emailprotected]

For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900

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Global Oncology Targeted Immunotherapy Market Report 2021: Rising Success of Gene and Cell Therapies, Gene Editing Techniques, and Novel Gene Delivery...

TCR2Therapeutics Inc., a clinical-stage Cell therapy company with a pipeline of novel T cell therapies for cancer patients suffering from solid tumors, and Arbor Biotechnologies, a Biotechnology company discovering and developing genetic medicines, have entered into a strategic research collaboration and non-exclusive license agreement focused on the further development of a defined set of allogeneic TRuC-T cell therapies.The collaboration leverages Arbors proprietary CRISPR Gene -editing technology, which is tailored to address the underlying pathology of genetic diseases, and TCR2s TRuC platform, which has demonstrated clinical activity in multiple treatment-refractory mesothelin-expressing solid tumor indications with its Lead autologous program gavo-cel.Our autologous TRuC-T cells have already established clinical activity in multiple difficult-to-treat solid tumors without being dependent on HLA, thus allowing our therapies to be used on the broadest patient population. We believe allogeneic TRuC-T cell therapies will further extend this impact by accelerating patient access while reducing manufacturing cost, said Garry Menzel, Ph.D., president and chief executive officer of TCR2 Therapeutics . The collaboration with Arbor enables us to evaluate multiple allogeneic candidates with novel enhancements and advance a lead candidate in 2022, consistent with our vision of continuing to innovate novel therapies for cancer patients suffering with solid tumors."The collaboration with TCR2allows us to leverage Arbor's proprietary discovery engine and gene editing technologies with an established Cell Therapy leader developing life-changing treatments for serious cancers, said Devyn Smith, Ph.D., chief executive officer of Arbor. This agreement reinforces the versatility and strength of Arbors platform and furthers our strategic vision of expanding the impact of Arbors gene editors through partnerships with leading organizations developing engineered cell therapies.Under the terms of the agreement, Arbor will receive an upfront cash payment and is also eligible to receive additional milestone payments based upon the successful achievement of development, regulatory and commercial milestones across a selected number of programs. In addition, TCR2will pay tiered royalties on future net sales on any products that may result from this collaboration.

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TCR2 Therapeutics, Arbor Biotechnologies Partner on TRuC-T Cell Therapies - Contract Pharma

CAMBRIDGE, Mass., Jan. 4, 2022 /PRNewswire/ -- Erbi Biosystems, developer of innovative, small-scale, automated, and high-performance cell culture systems, has closed a private round of funding totaling $4M. This latest financing will enable the company to expand operations to meet the growing demand for its Breez perfusion bioreactor product and develop extensions to enhance cell therapy applications.

The Erbi Breez bioreactor provides continuous media addition, cell retention and bubble-free gas diffusion through its True Perfusion technology, which is unique among existing small-scale bioreactors that can only approximate perfusion with intermittent media exchange. The Breez's fully automated, sterile, single-use system also includes online sensing and controls that are combined with easy-to-use software, delivering sophisticated feeding strategies, cell concentration, and media exchanges, resulting in performance that can exceed stirred tank and rocking bag reactors.

"Erbi has developed a compelling platform for small-scale cell culture," said Michael Chambers, Erbi investor and founding CEO of Aldevron. "In addition to its capabilities in bioprocessing, their platform enables testing of multiple replicates from a single sample of scarce patient cells - a unique and powerful capability that will accelerate cell therapy development."

"The Breez product has proven to be an effective scale-down tool for process development across a range of cell types," said Dr. Martin Madaus, former Chairman and CEO of Millipore. "The small volume, single-use, sterile and fully-automated format makes this an ideal platform for cell therapy manufacturing."

In 2021, Erbi Biosystems doubled its staff and relocated to a larger facility north of Cambridge,equipped with biolabs and cleanroom manufacturing space.

"We're growing quickly and expect to double again in 2022," said Dr. Michael Chiu, CEO of Erbi Biosystems. "Revenue from our growing list of Breez customers in the US, Europe and Asia is fueling our growth. This additional funding allows us to accelerate our pipeline of technology and product development projects which are focused on the needs of the cell therapy process development and GMP manufacturing."

Mr. Chambers and Dr. Madaus are both investors in the latest private round.

About Erbi Biosystems

Erbi Biosystems supports therapeutic drug manufacturers with tools to accelerate the developmentof their processes and therapies. Erbi's technology platform includes single-use, mL-scale cell culture, microfluidics, optical sensing, and integrated controls.

The Breez bioreactor is used across a range of applications and cell types, including media and cell-line development; intensified process and n-1; CAR-T process development; and non-mammalian cell culture. The small size, single-use device, and fully automated operation drastically reduce the time, media consumption and lab space required for process development. The mL-scale working volume and closed-sterile consumable format make it ideal when working with scarce and highly variable source materials, such as autologous therapy development.

The Erbi Breez system is available worldwide with sales and distribution in the US, EU, and China.

SOURCE Erbi Biosystems

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Erbi Biosystems Raises $4M to Provide Small-Scale Perfusion Bioreactors for Development and Manufacture of Cell Therapies - PRNewswire

MIAMI, Jan. 4, 2022 /PRNewswire/ --Dystrogen Therapeutics Corp. a clinical-stage company developing chimeric cell therapeutics to modify the course of rare diseases, today announced dosing of the first patient in its Phase1 Human PilotClinical Study of DT-DEC01, Dystrophin Expressing Chimeric cells, or DEC, for the treatment of Duchenne Muscular Dystrophy (DMD).

The Phase1 Human PilotStudy is a non-randomized trial enrolling boys between the ages of 5 and 18. The trial investigates the safety, tolerability, and efficacy of DT-DEC01. The pilot trial will enroll a total of 10 patients with DMD across 3 escalating doses. The study will also assess NorthStar Ambulatory Assessment (NSAA), range of motion (ROM), performance of upper limb (PUL), 6MWD, Muscle strength by myometry and 5-grade Lovett scale, and cardiac muscle evaluation by ECHO.

The first patient, a 6-year-old boy with deletion of exons 3-12, received an infusion of DT-DEC01 on November 26th, 2021. The patient tolerated the procedure well and was discharged home. At day 7, day 28, and 6 week follow up, the patient demonstrated no side effects from the treatment. Functional outcomes, including ECHO, demonstrated a non-clinically significant improvement when compared to pretreatment baseline. Subjective parent reported assessment of post - treatment daily activity level demonstrated a significant improvement when compared to pretreatment baseline at 6 weeks. Subjective therapist reported assessment of post - treatment quality of motion as well as patient performance during functional assessment demonstrated a significant improvement when compared to pretreatment baseline at 6 weeks. At 6 weeks, laboratory data demonstrated no signs of inflammation, infection, antibody formation, or other adverse systemic effects on end organs.

Screening and enrollment of patients is expected to continue over the course of the next 12 months in the European Union, with data from this trial expected once patients have been evaluated for one full year post-treatment.

"We are very pleased to begin dosing patients in our pilotstudy. This is the first time we will assess our chimeric cell therapy in Duchenne patients, which will provide us not only with safety data but could also give us an early look into the potential efficacy of this treatment in possibly halting disease progression and even partly restoring functional loss in boys with muscular dystrophy", saidKris Siemionow, MD, PhD Founder and Chief Executive Officer of Dystrogen Therapeutics.

"This is an important step toward bringing the promise of this novel technology to patients with serious diseases like DMD, and we are thrilled to be leading the effort in what we believe may be a fundamental change in the treatment of debilitating disorders", saidMaria Siemionow, MD, PhD Founder and Chief Scientific Officer of Dystrogen Therapeutics.

About DT-DEC01

DT-DEC01 is a chimeric cell therapy. The advanced therapeutic medicinal product (ATMP) is made using Dystrogen's proprietary cell engineering technology which creates a DEC cell. Clinically, DEC cells have been shown to express CD56 at significantly higher levels than myoblasts from Duchenne patients. DEC cells express favorable HLA characteristics which carries multiple advantages. In preclinical studies, DEC cells have also been shown to express clinically significant levels of dystrophin when compared to controls. DEC cell therapy demonstrated significant functional improvement in cardiac, diaphragm, and other skeletal muscle strength and associated function in preclinical trials. Because DEC therapy is designed to prevent triggering an immune system response, a major advantage of DEC therapy is that it does not require immunosuppression. The therapy is not associated with any genetic manipulation and therefore involves no risk of off target mutation, does not use viral vectors, and its use is not dependent on the genetic mutation of the DMD patient, thus making DEC a universal therapy for all DMD patients.

About Dystrogen Therapeutics Corp

Dystrogen Therapeutics is a clinical-stage life sciences company committed to developing therapies for rare genetic diseases. The company was founded based on the pioneering work of Prof. Maria Siemionow, a world-renowned scientist and surgeon who led the team that performed the first near-total face transplantation in the United States. Professor Siemionow's research focused initially on the creation of chimeric cells which have a role in modulation of the immune system's response to a transplant. This led to the development of Dystrophin Expressing Chimeric (DEC) cell therapy that is designed to prevent the immune system from attacking the chimeric cells. DECs are engineered cells and belong to a family of therapeutic technologies called Advanced Therapy Medicinal Products (ATMP). Using Dystrogen's patented cell engineering technology, DECs are made by combining a malfunctioning cell of the Duchenne patient with a normal, working cell from a healthy donor. This novel chimeric cell is composed of both the donor's and the recipient's cell structures but looks to the patient's immune system like his own cell and thus does not trigger an immune response while it functions (i.e. produces dystrophin) like the patients' normal cell. This offers a unique advantage and allows the patient's body and immune system to accept the chimeric cell without rejection. In such a way, Dystrogen has created dystrophin producing cells that can engraft in the patient's muscles (such as heart, diaphragm, skeletal muscles) and, as demonstrated in our research and related peer-reviewed publications, increase their dystrophin levels. Increased dystrophin levels have been shown to correlate with improved functional outcomes which was confirmed in preclinical studies of DEC.

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SOURCE Dystrogen Therapeutics Corporation

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Dystrogen Therapeutics Announces First in Man Dosing of Novel Chimeric Cell Therapy for Duchenne Muscular Dystrophy and Reports 6-week Clinical...

The biotech IPO market has been hot for the past few years, but it set all sorts of new records in 2021. A total of 399 companies priced IPOs in 2021, raising $142.5 billion, according to IPO research firm Renaissance Capital. Those figures made this year the busiest by deal count since 2000, and the biggest ever in terms of proceeds raised.

Healthcare, which includes biotech, was the busiest sector, accounting for 36% of the IPO activity, Renaissance said in its 2021 annual review. The biggest biotech IPO of the year belongs to Seattle cell therapy developer Sana Biotechnology, which was able to upsize an IPO that raised $588 million.

While Sanas IPO total is eye-catching, the sum is also notable because its the largest ever IPO for a company that has yet to reach the clinic. Not so long ago, biotech companies typically waited until they had clinical data before turning to the public markets. Even early dosing data reduces the investment risks.

Sana still doesnt have clinical data. What it does have is a cell and gene therapy technology and leadership from two former Juno Therapeutics executives who steered that company to a $9 billion acquisition by Celgene in 2018. Sana said it is preparing to file multiple investigational new drug applications in 2022 and 2023. That means investors must wait another year or more before the company produces data that could move the companys stock price.

Other cell therapy developers that went public this year include Lyell Immunopharma, Caribou Biosciences, Instil Bio, Talaris Therapeutics, and Achilles Therapeutics. Most of the biotechs in this group had reached at least early clinical testing at the time of their IPOs.

Recursion Pharmaceuticals had the second biggest biotech IPO of 2021. The Salt Lake City, Utah-based company raised $436.4 million to continue clinical development of drug candidates discovered by its AI platform. Similar to Recursion, Exscientia has an AI-based drug discovery platform that it uses to develop drug candidates for partners as well as its internal pipeline. The Oxford, U.K.-based company raised $304.7 million from its IPO. Absci, which uses its AI platform to produce proteins that become drug candidates for its pharmaceutical industry partners, raised $200 million in its stock market debut.

According to Renaissance, IPO filings increased for the third straight year; the 495 IPOs that were filed in 2021 through Dec. 20 is a more than 94% increase compared to the same period last year. The $142.5 billion raised is an 82.2% increase over last years total.

The year was also marked by a surge in companies going public by merging with special purpose acquisition companies (SPACs). Renaissance counted nearly 200 companies such deals. Biotech companies that went public in 2021 via SPAC deals include Nautilus Bio, Humacyte, Ginkgo Bioworks, and Pardes Biosciences.

As we head into a new year, the hot IPO market appears to be cooling off. While the market wont turn completely cold this winter, Renaissance doesnt think investors should expect a repeat of 2021s IPO activity.

Looking forward, we believe the 2022 IPO market will have a slow start and fail to match the past years record pace, but ample IPO candidates are ready to take the leap once conditions improve, the firm said.

Photo: Spencer Platt, Getty Images

Link:
Cell therapy, AI drug discovery highlight 2021's record year in biotech IPOs - MedCity News

Megan Olsen, MPH, principal at Avalere, discusses the role that value assessments are expected to play in the future pricing of gene and cell therapies.

Megan Olsen, MPH, principal at Avalere, discusses how the impact of small patient populations and other limitations may influence payer conversations about gene and cell therapies.

Transcript

What role will value assessments play in payer decisions on pricing for cell and gene therapies?

It's going to play an important role. And I think it already is today, to some extent. We've done some survey research with payers at Avalere and found that most payers are already doing their own valuations for products in this space. We've also seen other entities like [the Institute for Clinical and Economic Review] be referenced as a key stakeholder at informing valuation of various products in this space. When you have an introduction of a high cost potentially million dollar therapy, with the potential for the benefit over many years, it's going to get a lot of attention you'll want to understand what is the true value of the product relative to price. So, there are a lot of different frameworks under consideration for how to measure that and how to adapt various methodologies or framework specific to the cell and gene therapies, given the uniqueness of the asset.

There's a lot of evolving in the value-based space in the private market, but also in the policy sphere as well. We're seeing the new Biden administration emerge with its priorities in health care and I think value-based care is going to be at the top and we've heard references in the drug pricing arena on interest in tying prices to value in some way or leveraging or standing up a value assessment entity to really measure value and potentially have a role in informing price as well. So, I think a lot more to come there. And this whole swirling interest in innovative financing models is based on value and wanting to determine net pricing based on the value that the product is delivering to the patient, to the health care system, to the payer, etc. So, needing that data to to make those determinations but also using the data that is derived from these arrangements to inform future value conversations as well.

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Avalere Analyst Weighs in On How Value Assessments Will Influence Gene and Cell Therapy Pricing - AJMC.com Managed Markets Network

Sexton Cell Processing tools and CryoStor cGMP Freeze Media Incorporated in Cell and Gene Therapy Workflows

BOTHELL, Wash., Jan. 4, 2022 /PRNewswire/ -- BioLife Solutions, Inc. (NASDAQ: BLFS), a leading supplier of class-defining bioproduction tools and services for the cell and gene therapy and broader biopharma markets, today announced an extended collaboration with Seattle Children's Therapeutics, a venture at Seattle Children's, bringing cutting edge, curative technologies and therapies to defeat pediatric cancer and other diseases that impact children. The collaboration will focus on establishing best practices in biopreservation and closed-system manufacturing with the introduction and integration of Sexton's AF-500 for closed-system processing for cell therapy manufacturing and viral vector delivery that will be used in Seattle Children's Therapeutics new lentiviral vector manufacturing facility called VectorWorks.

BioLife Solutions New Logo 2021 (PRNewsfoto/BioLife Solutions, Inc.)

"This expanded collaboration is an exciting step for Seattle Children's Therapeutics as we work towards developing closed-system processes for cell therapy manufacturing," said Matt Selley, Director of GMP Manufacturing at Seattle Children's. "Lentiviral vector manufacturing is a critical component of the cell production process."

Seattle Children's has utilized BioLife's CryoStor cGMP freeze media for several years, as an optimized excipient for improved post-thaw viability and functional recovery of cells used in clinical applications and trials. As cell and gene therapy manufacturers move toward closed-system processing, it is vital that upstream critical excipients and ancillary materials, such as viral vectors, are manufactured and packaged in containers suitable for closed-system integration. While some upstream bioprocesses have been successfully automated, the final steps of downstream bioprocess, namely fill-finish, are often performed manually in open systems with associated risks of contamination and user error. Furthermore, current packaging for viral vector intermediates demands that therapy developers operate in higher grade environments due to the open nature of this manufacturing step.

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This continued collaboration will combine the Sexton off-the-shelf automated fill-finish system, AF-500, with Seattle Children's Therapeutics' expertise in viral vector manufacturing. Sexton's rapidly deployable AF-500 is capable of filling and sealing up to 560 CellSeal vials in 90 minutes. The goal is to utilize the AF-500 to initiate high throughput fill-finish of vector intermediates. Vectors will be filled into Sexton's proprietary vials, CellSeal and CellSeal Connect. CellSeal Connect builds on the original CellSeal cryogenic storage vial, which has been incorporated as the final drug packaging in commercial cell therapy products. The new version allows closed-system retrieval of intermediate products, such as viral vectors, thereby negating the need for therapy developers to operate in higher grade manufacturing suites for delivery of vectors and cargo.

The collaboration will result in detailed workflows, demonstrating the suitability of the CellSeal platform as a packaging container and closed system automation for viral vectors. It will include an assessment of the vial and fill system's usability and compatibility with high throughput fill-finish of viral vectors. In addition, post-fill activities such as high-density storage, shipping, distribution, thawing, and closed system retrieval will be assessed. As Seattle Children's Therapeutics will be the end-user of the viral vector product, the workflow resulting from the collaboration will cover the movement of viral vectors from the point of packaging to the point of transduction and patient administration, when applicable.

"The development of these closed connection processes in small volume aliquots has the potential to streamline this element of cell therapy manufacturing," said Sean Werner PhD, Chief Technology Officer, Cell Processing at Biolife Solutions. "Developing tools to meet the unique needs of this emerging industry is the best way to bring these life changing therapies to patients."

About BioLife SolutionsBioLife Solutions is a leading supplier of class-defining bioproduction tools and services for the cell and gene therapy and broader biopharma markets. Our tools portfolio includes our proprietary CryoStor and HypoThermosol biopreservation media for shipping and storage, the ThawSTAR family of automated, water-free thawing products, evo cold chain management system, high capacity cryogenic storage freezers, Stirling Ultracold mechanical freezers, SciSafe biologic storage services, and Sexton Biotechnologies cell processing tools. For more information, please visit http://www.biolifesolutions.com, http://www.scisafe.com, http://www.stirlingultracold.com, or http://www.sextonbio.com and follow BioLife on Twitter.

About Seattle Children's Seattle Children's mission is to provide hope, care and cures to help every child live the healthiest and most fulfilling life possible. Together, Seattle Children's Hospital, Research Institute and Foundation deliver superior patient care, identify new discoveries and treatments through pediatric research, and raise funds to create better futures for patients.

Ranked as one of the top children's hospitals in the country by U.S. News & World Report, Seattle Children's serves as the pediatric and adolescent academic medical center for Washington, Alaska, Montana and Idaho the largest region of any children's hospital in the country. As one of the nation's top five pediatric research centers, Seattle Children's Research Institute is internationally recognized for its work in neurosciences, immunology, cancer, infectious disease, injury prevention and much more. Seattle Children's Foundation works with the Seattle Children's Guild Association, the largest all-volunteer fundraising network for any hospital in the country, to gather community support and raise funds for uncompensated care and research. Join Seattle Children's bold initiative It Starts With Yes: The Campaign for Seattle Children's to transform children's health for generations to come.

For more information, visit seattlechildrens.org or follow us on Twitter, Facebook, Instagram or on our On the Pulse blog.

Cautions Regarding Forward Looking Statements

Except for historical information contained herein, this press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, statements concerning our extended collaboration with Seattle Children's and the results of such collaboration. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. These statements are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and assumptions that could cause actual results to differ materially from those described in the forward-looking statements, including among other things, those factors described in our risk factors set forth in our filings with the Securities and Exchange Commission from time to time, including our Annual Report on Form 10-K, Quarterly Reports on Form 10-Q and Current Reports on Form 8-K. We undertake no obligation to update the forward-looking statements contained herein or to reflect events or circumstances occurring after the date hereof, other than as may be required by applicable law.

At the CompanyTroy WichtermanChief Financial Officer(425) 402-1400twichterman@biolifesolutions.com

InvestorsLHA Investor RelationsJody Cain(310) 691-7100jcain@lhai.com

Cision

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SOURCE BioLife Solutions, Inc.

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BioLife Solutions Collaborates with Seattle Children's to Improve Viral Vector and Cell and Gene Therapy Manufacturing - Yahoo Finance