Sony has introduced a new cell isolation system, CGX10, which sorts cells at high speed and high purity in a closed system. The CGX10s design enables sorting of cells in a manner that is entirely closed off from the outside environment, making it possible to analyze and isolate cells while maintaining a sterile state. This is especially important in fields such as cell-based immunotherapy, which has been gaining recognition as a treatment for cancer and autoimmune disorders. To produce the cell therapies necessary for this treatment, there is an increasing demand for isolating cells with high purity and high viability in a sterile manner.

It is also expected that this product could be utilized in research for developing cell-based products for fields such as cell therapy and regenerative medicine. It can also contribute to the development and production of cell therapies in compliance with quality and production management regulations.

By introducing CGX10, Sonys life science business will expand from research applications to the field of cell therapies development and manufacturing, further contributing to the advancement of cell therapy.

Cell-based immunotherapy treatment is gaining recognition as a treatment for serious diseases, such as cancer, says Taisuke Kawasaki, senior general manager, life science division, Sony. Our cell isolation system CGX10 integrates closed multi parameter cell sorting technology, making it possible to accelerate the development of cell therapies. With this product, we are able to move from contributing to life science research to supporting the production and development of cell therapies. We will continue to contribute to society for people's safety and security by leveraging Sonys technologies in the life sciences field.

Until now, the main treatments for cancer have been surgery, radiation therapy, and anticancer drugs. However, in recent years there have been rising expectations for cell-based immunotherapy to be the new, fourth pillar of cancer therapies, says James Graziadei, CEO of the Sony biotechnology business. In addition to a treatment for cancer, cell-based immunotherapy is also recognized as a new treatment for autoimmune disorders such as diabetes and rheumatism. Alongside medical development, demand will continue to grow for cell sorting under the highest levels of sterilization and purity. With the introduction of CGX10, well aggressively support cell therapies.

Sonys CGX10 features the ability to use up to 10 parameters to isolate a single cell, and the signal is based on excitation from four lasers (405nm / 488nm / 561nm / 638nm) to detect target cells of interest. At analysis speeds of about 15,000 cells per second, it also ensures a purity level of about 97% and higher.

By controlling fluidics, cell damage is minimized, allowing for a high viability of sorted cells. Thus, it can increase the efficiency of the downstream production processes, such as cell culture and expansion for producing cell-based therapies. Furthermore, as this instrument uses fully disposable, single-use plastic tubing/components, any risk of cross-contamination between samples is avoided.

Sony is expected to begin deliveries of the CGX10 cell isolation system in fall 2022.

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Sony System for Use in Production of Advanced Cell Therapy Products From: Sony - Healthcare Packaging

CASE SUMMARY:

A 73-yearold woman presented with shortness of breath, productive cough, chest pain, fatigue, anorexia, and recent 18-lb weight loss. She had a history of hypertension and was a 45 pack-a-year smoker. An examination showed she had dullness to percussion and decreased breath sounds.

A chest x-ray showed a left hilar mass and a 5.4 cm left upper lobe mass. A chest/abdomen/pelvis CT scan showed a hilar mass with bilateral mediastinal extension and was negative for distant metastatic disease. PET scan showed activity in the left upper lobe mass and supraclavicular nodal areas and liver lesions. An MRI showed one small asymptomatic brain lesion. An interventional radiology biopsy of the liver led to a diagnosis of small cell lung cancer (SCLC) and the patients ECOG performance score was 1.

DISCUSSION QUESTIONS:

MELISSA L. JOHNSON, MD: Is there any other workup that you would recommend before you start therapy?

MICHEL E. KUZUR, MD: I am wondering if molecular testing has any benefit here?

JOHNSON: Well, because this is a SCLC patient, I do think about molecular profiling, but I would not wait to get that result back to start therapy.

Because we know that this patients diagnosis is SCLC based on the liver, and that she had CT scans of the head and an MRI, and because we do not check PD-L1 status in SCLC, I would say our workup is complete, and I think we are ready to start therapy.

For what proportion of your patients with extensive-stage SCLC is treatment initiated in the inpatient setting?

JOHNSON: Most of the participants responded that the majority of your patients with SCLC are not started in the inpatient setting, which is good, so they should all be treated in a frontline trial.

DISCUSSION QUESTIONS:

JOHNSON: I often ask sponsors if they could let me give 1 cycle in the hospital because sometimes SCLC is a rapid diagnosis, especially with virulent symptoms, so you do not have time to wait to get them to the clinic to give chemotherapy. And so if there is 1 kind of chemotherapy I know how to give in the hospital, it is carboplatin/etoposide. Does anyone disagree?

JEFFREY FRIEDMAN, MD, PHD: I agree, but I think that for a lot of us in sort of peripheral clinics like where I practice, the issue is that it is somewhere in between needing inpatient chemotherapy and can I wait 4 weeks to get their tissue sampled for whatever it takes to get them on trial? Typically, even though I am not treating them in the hospital, I am not waiting weeks. They are getting started on treatment in a few days or else things are just going to completely go off the rails, and so I think that is the bigger hurdle when it comes to trial accrual. Even just the ancillary imaging that might be needed for a trial is just not doable.

GREGG C. SHEPARD, MD: The other issue is that probably 90% or more of patients I meet are treated for the first cycle in the hospital but then they are discharged and sent to Dr Friedman at a peripheral clinic, so he can give them a second cycle as an outpatient. So he may be choosing 90%. He treats people in the outpatient setting, but I am treating them for their first cycle usually inpatient at Ascension Saint Thomas Hospital.

JOHNSON: If you have a couple more days, and you are waiting for Dr Shepard to refer a patient to come see you, for example, or you have seen a patient at TriStar Southern Hills Hospital and you have asked them to come to clinic, what is your approach to selecting treatment? What is the algorithm that you go through when you are thinking about a new SCLC diagnosis?

JACK W. ERTER, MD: I mean, I think luckily in this disease, for better or for worse, it is not like there are 17 different options I am weighing the pros and cons of and saying, "Are they are going to be a candidate for up-front EGFR-targeted therapy or similar." I mean, this carboplatin/etoposide chemotherapy plus immunotherapy. That is what they are starting on, unless they have 4 brain metastases, in which case they need radiation treatment first.

NANCY W. PEACOCK, MD: The other thing I think is, a lot of these people that have SCLC that lands them in the hospital; they do not have a ton of psychosocial support. They delayed coming in until they got very sick because they did not have the wherewithal to get there. They do not have the support at home. They come in from the hinterlands and often the first conversation we have with them is, "This is really a very difficult diagnosis. I can give you treatment." Even if their ECOG performance status is 3, it might not be worth treating them. Everybody says, "Oh, SCLC responds," but SCLC with a poor performance status does not respond very well. I think as we are being held more and more accountable for this oncology as a cure model, trying to keep expenses down and people out of the hospital is a decision that is being made more and more, but the people I see mostly are under-insured and [not receiving enough care].

JOHNSON: Those are also absolutely relevant topics for this patient population especially. I would say that carboplatin/etoposide remains the mainstay of treatment and luckily that is cheap in the inpatient setting. Obviously, the hospitalization costs more, but it is immunotherapy that adds the true cost. But that is a good point. In terms of treatment options, how are you counseling your patients?

KUZUR: In support of what Dr Peacock mentioned, sometimes the hospitalist has already convinced the patient to go on hospice, and they consult hospice and they consult me. I sometimes have to find the option and try to convince them to try some therapy. Dr Peacock is right. Some of these people are so sick you are not able to justify that you are going to get some active results with treatment.

DISCUSSION QUESTION:

JOHNSON: Does anybody want to explain why you think carboplatin or why you recommend cisplatin for a SCLC patient?

RYAN M. CARR, MD: I almost always use carboplatin, but I guess one reason I would consider cisplatin is if they have some degree of myelosuppression from some other reason to begin with, and we are able to tolerate the cisplatin, then I guess I could consider that. But almost everybody gets carboplatin with me.

ERTER: Yes. I rarely would use cisplatin and I do not think there is a big advantage in extensive-stage SCLC. I might consider it in limited curative therapy.

JOHNSON: Agreed. I think that is what our entire practice would say, and so this patient [would get] concurrent atezolizumab [Tencentriq] with carboplatin/etoposide and that was based on the IMpower133 [NCT02763579] data.

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Approaches and Obstacles to Frontline Treatment for Small Cell Lung Cancer - Targeted Oncology

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

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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...