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Berkeley Lights and the Jaime Leandro Foundation Announce the Discovery, Functional Characterization, and Recovery of a Patient-Derived T cell…

Posted: August 5, 2022 at 2:21 am

EMERYVILLE, Calif., Aug. 4, 2022 /PRNewswire/ -- Berkeley Lights, Inc. (Nasdaq: BLI), a leader in digital cell biology, and the Jaime Leandro Foundation for Therapeutic Cancer Vaccines (JLF), today announced the discovery, functional characterization, and recovery of a patient-derived T cell receptor sequence against a cancer neoantigen.

(PRNewsfoto/Berkeley Lights)

Together, Berkeley Lights and JLF were able to measure and identify T cells that were reactive against peptides used in a cancer vaccine that was administered to a patient to successfully stimulate an immune response against their advanced-stage pancreatic cancer leading to complete remission. The patient was treated under the supervision of physicians at Washington University in St. Louis.

The unprecedented speed of these results relied on the Berkeley Lights Beacon system. Applying the cell therapy development workflow, thousands of phenotypic measurements of single T cells were performed within one week. These measurements identified T cells that were cytotoxic and capable of secreting cytokines in response to antigen encounter, which were therefore predicted to be functional and subsequently sequenced at a single cell level. Cloning these T cell receptor sequences and expressing them in nave T cells enabled functional validation against an antigen of interest. This function-first measurement capability of the Berkeley Lights platform is a significant differentiator compared to frequency-based assessments of TCRs which result in functional validation bottlenecks, adding to a growing number of highly differentiated service offerings at Berkeley Lights.

"This unique workflow combines Berkeley Lights' core strength of functional analysis of live cells, along with the use of the new Biofoundry services organization to help customers accelerate their therapeutic discoveries in a faster, more scalable way," said Siddhartha Kadia, Ph.D., chief executive officer of Berkeley Lights. "We look forward to continuing to work with JLF and their partners in evaluating immune response to cancer vaccines, mapping TCRs to neoantigens and ultimately supporting this important work to save patient lives."

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The JLF has the mission of providing personalized neoantigen cancer vaccines for appropriate patients who have advanced cancers and seek compassionate use treatment.

"The data is exceedingly clear that, together, we have successfully identified a functional, patient- and antigen-specific TCR validating both our treatment methodology and the Berkeley Lights technology for personalized cancer vaccines compared to conventional approaches," said William Hoos, president of the Jaime Leandro Foundation for Therapeutic Cancer Vaccines. "This is an exciting advancement in our mission."

From the clinical team, Dr. William Gillanders, professor of surgery at Washington University School of Medicine, commented:

"The Berkeley Lights technology can take immune monitoring to the next level by evaluating multiple quantitative measurements of live cell behavior and recover that cell for molecular characterization. This is going to be tremendously useful for the field by providing a much deeper dataset to further understand cancer vaccines and predict clinical outcomes as well as to serve the growing need of combination therapies where TCR-T can be utilized."

1Daniel A King, Amber R Smith, Gino Pineda, et al. Complete remission in a patient with widely metastatic HER2-amplified pancreatic adenocarcinoma following multimodal therapy informed by tumor sequencing and organoid profiling. medRxiv 2021.12.16.21267326; doi: https://doi.org/10.1101/2021.12.16.21267326

About Berkeley Lights

Berkeley Lights is a leading digital cell biology company focused on enabling and accelerating the rapid development and commercialization of biotherapeutics and other cell-based products for our customers. The Berkeley Lights Platform captures deep phenotypic, functional, and genotypic information for thousands of single cells in parallel and can also deliver the live biology customers desire in the form of the best cells. Our platform is a fully integrated, end-to-end solution, comprising proprietary consumables, including our OptoSelect chips and reagent kits, advanced automation systems, and application software. We developed the Berkeley Lights Platform to provide the most advanced environment for rapid functional characterization of single cells at scale, the goal of which is to establish an industry standard for our customers throughout their cell-based product value chain.

Berkeley Lights' Beacon and Lightning systems and Culture Station instrument are FOR RESEARCH USE ONLY. Not for use in diagnostic procedures.

Forward-Looking Statements

To the extent that statements contained in this press release are not descriptions of historical facts regarding Berkeley Lights or its products, they are forward-looking statements reflecting the current beliefs and expectations of management. Such forward-looking statements involve substantial known and unknown risks and uncertainties that relate to future events, and actual results and product performance could differ significantly from those expressed or implied by the forward-looking statements. Berkeley Lights undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties relating to the Company's growth and continual evolution see the statements in the "Risk Factors" sections, and elsewhere, in our filings with the U.S. Securities and Exchange Commission.

Cision

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Valo Therapeutics Advancing Plans for Oncolytic Virus Treatments With Tailored Immunogenic Peptides – Precision Oncology News

Posted: August 5, 2022 at 2:21 am

NEW YORK Valo Therapeutics has reimagined oncolytic virus treatments by adding an exterior coating of tumor-specific peptides designed to stimulate an immune response against the tumor.

This enhancement, according the company, has opened development pathways for personalized therapy based on its PeptiCRAd technology.

PeptiCRAd uses electrostatic interactions to attach immunogenic peptides with a positively charged lysine tail to the negatively charged outside surface of a virus or bacteria. It was developed at the University of Helsinki, which spun out Valo in 2016.

The Finnish firm's lead product, PeptiCRAd-1, is an engineered adenovirus coated with NY-ESO-1 and MAGE-A3 tumor antigens genetically modified for increased tumor cell infection, high viral replication within the tumor, and increased immunogenicity. It also carries two co-stimulatory molecules as transgenes. One encodes a CD40 ligand to drive CD8+ T-cell responses. Another encodes an OX40 ligand for increased clonal expansion and survival of CD8+ T cells, and formation of a larger pool of memory T cells.

In nature, oncolytic viruses infect and kill cancer cells. Their potential as therapeutics has been harnessed through engineering of oncolytic viruses to have specific cancer-fighting properties. The US Food and Drug Administration approved Amgen's Imlygic (talimogene laherparepvec) for melanoma as the first oncolytic viral immunotherapy in 2015, and a number of others are in clinical trials, including some that use biomarker strategies and others in which oncolytic viruses are being combined with autologous CAR T-cell treatments.

Examples include a Phase I trial of the oncolytic virus Vaxinia (CF33-hNIS) and Merck's Keytruda (pembrolizumab) in metastatic solid tumors started by Imugene and City of Hope in May; and Phase I and Phase II trials by Vyriad of an attenuated measles virus and vesicular stomatitis virus alone and in combination with checkpoint inhibitors. The biomarker of interest in the City of Hope/Imugene and Vyriad trials is PD-L1 expression. Meanwhile, Mustang Bio is testing out an oncolytic virus with an autologous CAR T-cell therapy in brain cancer patients.

According to Valo CEO Paul Higham, what differentiates Valo's approach from others is the peptide coating, which gives the virus additional vaccine-like properties on top of the oncolytic effect of the virus. Because the peptides are attached weakly to the virus exterior by electrostatic charge rather than chemical bonds, they are released in the tumor environment, stimulating an immune response.

"Oncolytic viruses in the past stimulated a strong immune response. But most of that has been directed against the virus itself, not against the tumor," said Higham. "We're really shifting the focus of the immune system away from the virus and onto the tumor."

In preclinical research, the Helsinki-based company has shown that a PeptiCRAd virus targeting MAGE-A1 eradicated tumors in humanized mice with melanomas and increased the population of MAGE-A1-specific CD8+ T cells. "We've seen in our preclinical work, we have something close to a 90 percent response rate in the mice that we've treated," said Higham, adding that the effect is increased when a PeptiCRAd therapy is combined with checkpoint inhibitors.

Higham said Valo has also conducted experiments in which two identical tumors are grafted onto a mouse, one on the right side and one on the left, and PeptiCRAd therapy is injected into one of the tumors. Researchers observed an expected effect in the PeptiCRAd-injected tumor, but they also saw a "really strong effect" on the untreated tumor on the other side of the body.

"The only way you can really explain that is that we've stimulated the immune system in the right way and the relevant immune cells mainly T cells have gone throughout the mouse's body, identified that there's a tumor on the other side, and attacked that tumor," Higham said. Those results from mouse studies suggest that in humans this therapy could be active against metastases via a systemic immune reaction, even when only the primary tumor is treated.

Valo is hoping to see this effect in an upcoming Phase I clinical trial, in which it is combining PeptiCRAd-1 with Keytruda in 15 patients with melanoma, triple-negative breast cancer, and non-small cell lung cancer. Higham said patients in the study will receive four injections of PeptiCRAD-1 into their tumors followed by checkpoint inhibitor therapy "to take down any barrier there may be to T cells getting in and killing tumor cells." Researchers will measure changes in immune cells throughout the body indicating they are recognizing tumor antigens as well as patients' responses to therapy.

Valo, which is currently raising money for the study, has started recruiting patients at sites in Germany and expects to begin dosing patients before year end. The trial will report data before the end of 2023. "Within less than 18 months from now, we hope to have the full results of the study available," Higham said.

The company's acquisition of the PeptiCHIP platform from the University of Helsinki last month adds personalization capability to its development plans for its pipeline of PeptiCRAd therapies. PeptiCHIP combines microfluidic chip technology with a software algorithm to identify antigens from a tumor. Valo plans to develop individualized, n-of-1 therapies by using PeptiCHIP to test a patient's tumor for antigens and treating the patient with a PeptiCRAd therapy targeted to those antigens specifically.

That could happen in one of two ways. First, the company could create a bespoke therapy based on the patient's tumor antigen profile. A second option, Higham said, might be to create a library of the most common peptide antigens patients with a specific tumor type tend to have and cross-match an individual's PeptiCHIP analysis with that library to find an off-the-shelf PeptiCRAd therapy.

While a similar analysis using conventional methods might take two or three days, Higham said that Valo hopes to develop a method that will complete the tumor antigen analysis in two to three hours.

Toward that end, Higham said Valo's Phase I study would serve as proof of concept to show that the PeptiCRAd platform works. Once that's established, the company will expand into personalization strategies. That would put the start of clinical trials at least two years out, Highman estimated.

Another possibility being explored by Valo is repeating the antigen screening periodically during treatment and in follow-up in order to adapt the therapy to changes in the tumor, which often leads to loss of effectiveness of immunotherapies over time. "The targets we initially choose may not, six months or a year down the line, be the most relevant targets," said Higham. "We may have the adaptability to flex with the tumor as it changes."

While Valo is able to go after a variety of cancers with PeptiCRAd therapies, one limitation is that the tumor must be accessible to inject with a needle. Higham said that liver cancer and bladder cancer could be future areas of study for the company, but that with personalized therapy, the type of cancer "becomes a little bit irrelevant because we're treating individual tumors in a particular patient. If we can get biopsy sample from them, we can create a therapy."

Comparing a personalized PeptiCRAd therapy to autologous CAR T cell-therapy, which is also an n-of-1 treatment, Higham noted that adapting the therapy to changes in the tumor would require starting over, which would be expensive and time-consuming. "CAR T therapies can cost up to $400,000," Higham said. "Our therapy would be well below $100,000 because we're using a simpler, off-the-shelf oncolytic virus. That doesn't change. We just change the antigens."

In terms of regulation, Higham said the pathway for its Phase I PeptiCRAd product is "fairly straightforward." The company has permission from the German Regulatory Agency to proceed with its Phase I trial, and oncolytic viruses are an established mode of therapy. "The trickier thing is the adaptability we want to build in regarding the personalized therapy, and there may be a number of ways of doing that," Higham said.

That's where the use of a library may be helpful. "What we'd aim to do is have the peptides in the library approved by the regulatory authority so we're then free to select down from that library at any time and not have to seek individual regulatory approval for each patient or each therapy."

Because the peptide-coating technology is applicable to any oncolytic virus, Higham said that Valo is talking with other oncolytic virus companies about potential partnerships to apply the PeptiCRAd technology to their viruses. "It's a way our technology platform can [move] forward into Phase II and III studies very quickly, because we'd simply be enhancing what's already there," Higham said.

The firm is eyeing opportunities to work with companies developing checkpoint inhibitors or cell-based therapies, as PeptiCRAd therapies can potentially be combined with those, as well as with companies in need of a platform for validating neoantigens.

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DermaPrecise(TM) Injection System Shown to Produce Consistently High Cell Count and Viability for Cell Therapy Injections – Yahoo Finance

Posted: August 5, 2022 at 2:21 am

Independent Testing Performed by Innovacell Demonstrates the DermaPrecise Control Leads to Unparalleled Post-Injection Cell Survival and Viability Consistency

VANCOUVER, BC / ACCESSWIRE / August 2, 2022 / RepliCel Life Sciences Inc. (OTC PINK:REPCF)(TSXV:RP)(FRA:P6P2) ("RepliCel" or the "Company"), a company developing next-generation technologies in aesthetics and orthopedics, announced today it has received test results showing that cells injected through the DermaPrecise Injector maintained >99% viability.

Innovacell, a cell therapy company headquartered in Tokyo, Japan with a manufacturing facility in Innsbruck, Austria, tested RepliCel's DermaPrecise Injection System to measure for loss of cells and/or cell viability when injected through the system. Study results showed consistent cell viability over 99% when cells were injected through the DermaPrecise Injection System. Equally important as it relates to clinical practice, cells remained viable in the device for up to 90 minutes after which cells injected through the DermaPrecise Injector still maintained >98% viability with a simple pre-injection "rotation" of the injector wand to reverse any sedimentation.

Alternative dermal injection systems are either manual - and thus inherently inconsistent - or involve injection forces which are harmful to cells. Traditional manual injection techniques do not control for injection variability in shear stress, depth, dose, speed, or injection force. All these factors are known to influence cell viability and have a likely impact on clinical outcomes and yet, surprisingly, remain uncontrolled in many clinical cell therapy studies.

The DermaPrecise is an electronically controlled dermal injector with highly precise, pre-fixed volume and depth parameters which dictate exact consistency of every injection. Additionally the DermaPrecise system is programmable to ensure injection delivery speed does not exceed the shear force harmful to cells after factoring in cell density, needle gauge, and patient tissue density. Furthermore, the DermaPrecise injects cells while the needle is being withdrawn, leaving the cells behind in the channel created by the needle, thus additionally reducing the amount of stress imposed upon injected cells.

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RepliCel Life Sciences, Inc., Tuesday, August 2, 2022, Press release picture

Given the system's control over injection depth, the injection can be optimized to deliver cells where they need to be to have an optimal clinical effect. DermaPrecise thus assists product developers and clinicians in controlling for delivery variability as an influence on clinical outcome variability.

"One of the things we've always believed about RepliCel's RCH-01 cell therapy, for example," stated R. Lee Buckler, President and CEO of RepliCel Life Sciences which has developed and owns all rights to the DermaPrecise product line, "is that it will be critical to optimizing the treatment outcome to consistently deliver the cells in the entire hair-thinning region of the scalp at the depths in which the hair follicle bulbs exist. What is exciting about the results of the recent study done at Monasterium Laboratory, when combined with the results of this Innovacell study, is that we are now clearly showing the ability to consistently inject a 100L volume using a 9-needle head in a 1cm2 array at 8mm maximum needle travel with the injection performed during needle withdrawal and, as per the image attached to this release demonstrates, ensure deposition in the mid-to-lower dermis and upper subcutaneous fat layer, at depths consistent with the location of hair follicle bulbs in androgenetic alopecia affected scalp. We believe this will be enormously valuable to our RCH-01 program by eliminating two important variables which are known to be linked to the success of clinical outcomes."

"The test results show that DermaPrecise has the potential to greatly improve consistent post-injection cell viability in the delivery of cells injected into dermal or subcutaneous tissue. Improved, controllable, consistent, validated cell-delivery tools are required to optimize cell therapy outcomes for patients and the companies developing these valuable therapies," stated Kevin McElwee, RepliCel's Chief Science Officer.

Study Design

RepliCel's non-bulbar dermal sheath (NBDS) cells (which form the basis of the Company's RCS-01 and RCS-01 products) were cultured and placed into a standard injection solution, injected through the DermaPrecise injector using the system's 9-needle consumable, cells were collected post-injection and stained, then processed through a flow cytometer to count the post-injection cell survival rate.

Market Demand for Controlled Delivery Systems

In a 2017 publication in the Nature Partner Journal, Regenerative Medicine*, authors from the University of Nottingham (UK) and University of Pittsburgh (PA, USA), stated:

"existing cell-delivery approaches have shown limited success, with numerous studies showing fewer than 5% of injected cells persisting at the site of injection within days of transplantation."

The authors, in exploring a variety of reasons for this loss of post-injection cell viability including variability in mechanical injection force used by manual injectors, the damage subjected to cells by shear force when injection pressures are too high, and a lack of focus on controlling for cell delivery protocols or technologies, concluded:

"There is a growing recognition that conventional needle-based cell transplantation tools have considerable inadequacies that may affect clinical translation."

"An integrated approach to the evaluation of cell-delivery success is needed to improve the assessment of delivery efficacy and to allow for sound interpretations of clinical results. Improved cell-delivery tools are also required to streamline the delivery of cell-based therapeutics from the donor to the patient without compromising quality. Finally, pre-clinical planning and testing of the desired administration protocol with cell-type specificity is essential to achieve good clinical trial design."

* Amer MH, Rose FRAJ, Shakesheff KM, Modo M, White LJ. Translational considerations in injectable cell-based therapeutics for neurological applications: concepts, progress and challenges. NPJ Regen Med. 2017 Aug 10;2:23. doi: 10.1038/s41536-017-0028-x. PMID: 29302358; PMCID: PMC5677964. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5677964/.

About Innovacell AG

Innovacell AG was founded in 2004 in Innsbruck, Austria and is, since 2021, a 100% subsidiary of Innovacell K.K. in Tokyo, Japan. Innovacell is a biotechnology company focused on developing, producing and marketing cell therapies for treating fecal and urinary incontinence. Innovacell has also developed technologies for isolating, multiplying, using and delivering muscle cells. Patents for these technologies have already been granted or their applications filed. Innovacell aims at marketing the products it has developed in the five key European countries (Germany, France, Italy, Spain and the United Kingdom) as well as in Japan and the USA. The goal is to become a global leader in the field of regenerative medicine with a focus on innovative, personalized cell therapies. Innovacell also performs select contract development and manufacturing services for clients such as RepliCel Life Sciences, Inc.

About the DermaPrecise Injector Product Line

The DermaPrecise Injector platform is an electronic injection system which will bring new levels of control over intra-dermal or subcutaneous injections where precision of depth, dose and/or delivery matters.

About RepliCel Life Sciences

RepliCel is a regenerative medicine company focused on developing cell therapies for aesthetic and orthopedic conditions affecting what the Company believes is approximately one in three people in industrialized nations, including aging/sun-damaged skin, pattern baldness, and chronic tendon degeneration. These conditions, often associated with aging, are caused by a deficit of healthy cells required for normal tissue healing and function. These cell therapy product candidates are based on RepliCel's innovative technology, utilizing cell populations isolated from a patient's healthy hair follicles.

The Company's cell therapy product pipeline is comprised of RCT-01 for tendon repair, RCS-01 for skin rejuvenation, and RCH-01 for hair restoration. RCH-01 has been the subject of successful safety and dose-finding clinical studies and is now the subject of its third clinical study evaluating efficacy for the treatment of male and female hair loss due to androgenetic alopecia. This ongoing study is being funded by Shiseido Company Limited pursuant to a license agreement which has now been terminated, but is the subject of an arbitration regarding Shiseido's rights to the product for Asia. RepliCel maintains the undisputed rights to RCH-01 for the rest of the world. RCT-01 and RCS-01 are exclusively licensed in Greater China to YOFOTO (China) Health Company. RepliCel and YOFOTO are currently co-developing these products in China. RepliCel maintains the rights to these products outside of Greater China.

RepliCel has also developed a proprietary injection device (DermaPrecise) and related consumables, which is expected to improve the administration of its cell therapy products and certain other injectables. YOFOTO has exclusively licensed the commercial rights for the DermaPrecise device and consumables in Greater China for dermatology applications and is expected to first launch the product in Hong Kong upon it being approved for market launch in either the United States or Europe. Please visit replicel.com for additional information.

Notable Facts:

RepliCel's three cell therapy products have now been tested in over 100 patients in four countries on three continents.

RepliCel now has key strategic regional partners each of which are now investing heavily in the further clinical testing and development of RepliCel's products for their markets. Data from each of the clinical programs will strengthen the product development initiatives for RepliCel and its other partners worldwide.

For more information, please contact:Lee Buckler, CEO and President604-248-8693info@replicel.com

Neither TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release.

SOURCE: RepliCel Life Sciences, Inc.

View source version on accesswire.com: https://www.accesswire.com/710326/DermaPreciseTM-Injection-System-Shown-to-Produce-Consistently-High-Cell-Count-and-Viability-for-Cell-Therapy-Injections

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DermaPrecise(TM) Injection System Shown to Produce Consistently High Cell Count and Viability for Cell Therapy Injections - Yahoo Finance

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Treating Multiple Myeloma Following Quadruplet Induction Therapy and ASCT – Targeted Oncology

Posted: August 5, 2022 at 2:21 am

CASE SUMMARY

A 54-year-oldwoman presented with Revised International Staging System stage II multiple myeloma, based on evaluations that showed a hemoglobin level of 7.0 g/dL, 2-microglobulin of 6 mg/dl, albumin 3.2 g/dL, calcium 11.3 mg/dL, lactate dehydrogenase of 200 U/L, and creatinine clearance of 45 mL/min. Bone marrow showed 22% clonal plasma cells. Serum kappa free light chains were 24 mg/dL. She had no cytogenetic abnormalities and an ECOG performance score of 1. A PET/CT scan showed multiple bone lesions in the vertebrae. She had no extramedullary disease. She was diagnosed with IgG-kappa myeloma and was considered transplant eligible.

Daratumumab (Darzalex), bortezomib (Velcade), lenalidomide (Revlimid), and dexamethasone (Dara-VRd) induction therapy was initiated. She achieved a very good partial response (VGPR) post induction therapy. She underwent stem cell mobilization and 2 months later underwent autologous stem cell transplant (ASCT). Her post-ASCT response was a VGPR.

DISCUSSION QUESTIONS

CAITLIN COSTELLO, MD: This patient did get a quadruplet regimen with dara-VRd. She achieved a VGPR post-induction, had stem cells mobilized, underwent her transplant, and post-transplant her response is a VGPR. What would you do next?

THOMAS DEKKER, MD: Consolidation with CAR [chimeric antigen receptor] T-cell therapy.

COSTELLO: With CAR T cell, sure. Youre going for it; I like it. This patient is post-transplant, they have a VGPR. The GRIFFIN study [NCT02874742] would give these patients consolidation with dara-VRd.

PREETI CHAUDHARY, MD: I would not do CAR T-cell therapy.

COSTELLO: What would you do?

CHAUDHARY: In my opinion, in multiple myeloma, patients do a maximum of 11 months with CAR T-cell therapy. It has a good response, but I dont think thats sustainable.

COSTELLO: I appreciate throwing ideas out there. That is not something we have an option to do right now. Its an interesting option, and something we can talk about; but yes, I agree with you. I think for the meantime, short of trials that are looking at doing CAR T-cell therapyparticularly for those patients who have not had an adequate response to transplant or consolidation, or patients who relapse shortly after their transplantI think the standard of care as it stands now is doing consolidation or trying to find a maintenance regimen to get them to minimal residual disease [MRD] negativity.

With all that being said, what are we going to do now for these patients? Weve talked about what these transplant eligible patients are getting consolidation and maintenance; weve talked about maintenance approaches for these patients who get quadruplets, to put them on doublets. Seeing all those deep response rates, is anyone getting cold on transplants? If we are going to get 90% remission rates, does anyone reconsider the role of transplant here?

PAMELA MIEL, MD: I dont make that call, meaning I still send patients to the transplant doctors to see if theyre going to proceed with the transplant or not. But, if theyre transplant eligible, they get referred.

COSTELLO: As a transplanter, I thank you for that. We want to see these patients, make the decisions, have the discussion with the patients so we can look at their risk/benefit profile, and understand their responses to their current therapy. So, please still send them in their third cycle, if not earlier, so we can have those discussions and make plans.

There are a lot of maintenance regimens that are out there, and different things to choose from; a whole other conversation in and of itself. Lenalidomide is the mainstay where we have an overall survival benefit, where we dont have it in any other maintenance regimens.1 But it does allow for the option of continued doublets. I think we will soon see daratumumab and lenalidomide as a doublet get added on to that maintenance therapy once we have some of these randomized trials that are going on that show the continued benefit of patients to get daratumumab in the maintenance setting if they did not receive it in the up-front setting.

DISCUSSION QUESTION

How likely are you to change your practice with respect to management of transplant eligible newly diagnosed myeloma?

DEKKER: I already use quadruplet.

MILAN SHETH, MD: I feel that we still need a lot of long-term data to get a better sense of what it is that were achieving with the quadruplet therapy. Im still not convinced everybody needs quadruplet therapy. I think somebody else had already said that we know were going to get better responses because were using great drugs, but do we need to use everything up-front? I feel like theres still a lot of unanswered questions here.

MIEL: Ive been wanting to put patients on quadruplet treatment. I dont know if you know Nina Shah, MD, over at UC San Francisco, but Ive attended some of her talks, and shes pushing for the quadruplet treatment. The only thing that changed my mind was that when I spoke to the transplant doctor at UC San Diego, he said, If its not very high-risk disease, Id go with VRd [bortezomib, lenalidomide, and dexamethasone]. So, I put the patient on VRd. But I probably would want to put someone on dara-VRd, given the chance.

COSTELLO: Yes. I think that my takeaway from the data has been that we would, of course, love long-term data to come out, butwe have to wait a long time for it. While were waiting for some of these phase 3 studies to go on, which are happening now to look at real randomized data, to play out, I find that this is just too intriguing to not do quadruplets for everybody now.

Since [these data were presented at [the 2021 American Society of Hematology annual meeting], Ive transitioned just about everyone whos at least transplant eligible over to quadruplet regimens now.2 Any patients who are on the fence, where Im not sure if theyre going to be eligible for transplant, I still will try and give them the benefit of a quadruplet regimen, and very quickly drop the bortezomib if I get worried about them, and end up with dara-Vd [daratumumab, lenalidomide, dexamethasone]. But I think these MRD negativity rates are just too good, and if that is going to be the true surrogate end point that were all aiming for, dara-VRd has been my go-to for the last 6-plus months or so for these patients, until someone tells me otherwise.

References

1. Ho M, Zanwar S, Kapoor P, et al. The effect of duration of lenalidomide maintenance and outcomes of different salvage regimens in patients with multiple myeloma (MM).Blood Cancer J. 2021;11(9):158. doi:10.1038/s41408-021-00548-7

2. Laubach JP, Kaufman JL, Sborov DW, et al. Daratumumab (DARA) plus lenalidomide, bortezomib, and dexamethasone (RVd) in patients (pts) with transplant-eligible newly diagnosed multiple myeloma (NDMM): updated analysis of GRIFFIN after 24 months of maintenance. Blood. 2021;138(Suppl_1):79. doi:10.1182/blood-2021-149024

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Treating Multiple Myeloma Following Quadruplet Induction Therapy and ASCT - Targeted Oncology

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World ROR1-Targeted Therapy Market Research Report 2022 with Profiles of Companies Active in the development of Anti-ROR1 Therapy Candidates – Yahoo…

Posted: August 5, 2022 at 2:21 am

DUBLIN, Aug. 3, 2022 /PRNewswire/ --The "ROR1-Targeted Therapy: Target Expression Profile, Safety & Efficacy of Drug Modalities, Pipeline Review, and Competitive Landscape Analysis" report has been added to ResearchAndMarkets.com's offering.

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This report evaluates Receptor tyrosine kinase-like Orphan Receptor 1 (ROR1) from an industry perspective for its suitability as a tumor-specific target for cancer therapy based on its expression profile and preclinical and clinical safety and efficacy data of the various drug modalities employed for discovery and development of ROR1-targeted therapy candidates.

The report has identified the players in the field and presents a competitive landscape analysis of stakeholders and a pipeline review based on the specific profiles of drug candidates and companies active in the field. The report includes information about business transaction in the field, such as acquisitions, partnerships & collaborations and licensing deals. Furthermore, the financial background and situation of the key players is described.

Receptor tyrosine kinase-like orphan receptor 1 (ROR1) is a type I transmembrane protein that is physiologically expressed in early embryogenesis and plays a critical role in organogenesis. Expression of ROR1 attenuates rapidly after embryonic development, becoming virtually undetectable on post-partem tissues, with the exception of a few B cell precursors. In contrast, ROR1 is expressed on a variety of cancers, particularly those that are less differentiated, and is associated with early relapse after therapy or metastasis.

Because of its tumor-specific expression and potential functional significance, ROR1 has become of interest as a target for various drug modalities, especially with enhanced effector function. The most advanced molecules is currently being evaluated in potentially registrational phase II/III studies, but the majority of programs are in late preclinical and early clinical development, thus still offering opportunities for improvements.

This report is based information retrieved from proprietary database, clinical trial registries, abstracts, presentations and posters from scientific meetings as well as full publications, from company websites, press releases, SEC filings, investor and R&D presentations.What will you find in the report:

The scientific rationale for ROR1-targeted therapies based on target characteristics and its differential expression profile;

Preclinical safety of ROR1-targeted therapy candidates;

Clinical experience and proof-of-concept with ROR1-targeted drug modalities;

Clinical indications suitable for development of ROR1-targeted therapies and their patient populations;

The competitive landscape of ROR1-targeted drug modalities in development;

Specific profiles of ROR1-targeted drug modalities; and

Profiles of companies active in the development of anti-ROR1 therapy candidates.

Key Topics Covered:

Executive Summary

1 Target Background: Structure & Function

2 Target Antigen Expression Profile2.1 Hematologic Malignancies2.2 Various Solid Tumors2.3 Lung Cancer2.4 Pancreatic Cancer2.5 Colorectal Cancer2.6 Breast Cancer2.7 Ovarian Cancer

3 Preclinical Safety of ROR1-Targeted Therapy Candidates

4 Clinical Experience with ROR1-Targeted Drug Modalities

5 Clinical Indications & Patient Populations5.1 Chronic Lymphocytic Leukemia5.2 Mantle Cell Lymphoma5.3 Diffuse Large B-Cell Lymphoma5.4 Triple Negative Breast Cancer

6 Competitive Landscape & Drug Modalities6.1 ROR1-Targeted Antibody Drug Conjugates6.2 T-Cell EngagingAnti-ROR1 Antibodies6.3 Adoptive Cell Therapy with ROR1-Targeted CAR T-Cells and NK Cells6.4 ROR1 Small Molecule Inhibitor

7 Drug & Cell Therapy Candidate Profiles7.1 Naked Antibody7.2 Antibody-Drug Conjugates7.3 T-Cell Engaging Bispecific Antibodies7.4 CAR T-Cells & NK Cells7.5 Small Molecule Inhibitor

8 Company Profiles

9 References

For more information about this report visit https://www.researchandmarkets.com/r/1xld2f

Media Contact:

Research and MarketsLaura Wood, Senior Managerpress@researchandmarkets.com

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Global Genome Editing Technologies market is projected to grow at a CAGR of 15.96% by 2032: Visiongain Reports Ltd – Yahoo Finance

Posted: August 5, 2022 at 2:20 am

Visiongain Reports Ltd

Visiongain has published a new report entitled Global Genome Editing Technologies Market, (COVID-19 Impact Analysis):- Market Segment by Type (CRISPR, TALEN, ZFN, Antisense, Others), Market Segment by Application (Cell Line Engineering, Genetic Engineering, Diagnostic applications, Drug discovery & development, Others), Market Segment by End-user (Biotechnology companies, Pharmaceutical companies, Academic & Government Research Institutes, Others) plus COVID-19 Impact Analysis and Recovery Pattern Analysis (V-shaped, W-shaped, U-shaped, L-shaped), Profiles of Leading Companies, Region and Country.

The Global Genome Editing Technologies market is estimated to be valued at US$ 4,225.48 million in 2022. The market is projected to reach a market value of US$ 18,570.41 million by 2032. We predict strong revenue growth through to 2032

Download Sample here -https://www.visiongain.com/report/genome-editing-technologies-market-2022/#download_sampe_div

How has COVID-19 had a positive impact on the Genome Editing Technologies Market?

The COVID-19 pandemic has prompted large pharmaceutical and biotechnology firms, as well as genomic market participants, to engage in vaccine research and development. The rising need for vaccines and potential antiviral candidates has propelled genome editing/engineering technologies to the forefront. CRISPR technology was successfully used to provide rapid diagnostic tests for COVID-19, leading in its first FDA clearance (MD, USA). Many firms are rushing to cover the ever-widening market vacuum generated by reagents for PCR-based COVID-19 tests running out and testing capacity dwindling while rapid diagnostic tests are now being developed for wider clinical use. In other areas, researchers have considered CRISPR as a viable therapeutic, utilizing its targeted enzymatic activity to degrade SARS-CoV-2 RNA and halt viral replication."

How will this Report Benefit you?

Visiongains 414-page report provides 154 tables and 279 charts/graphs. Our new study is suitable for anyone requiring commercial, in-depth analyses for the Global Genome Editing Technologies Market, along with detailed segment analysis in the market. Our new study will help you evaluate the overall global and regional market for Genome Editing Technologies. Get a financial analysis of the overall market and different segments including gene editing technologies, applications, end-users, and company size, and capture a higher market share. We believe that there are strong opportunities in this fast-growing Genome Editing Technologies market. See how to use the existing and upcoming opportunities in this market to gain revenue benefits in the near future. Moreover, the report will help you to improve your strategic decision-making, allowing you to frame growth strategies, reinforce the analysis of other market players, and maximize the productivity of the company.

Story continues

What are the Current Market Drivers?

Rising investments in Genome Editing Technologies Governments of numerous nations throughout the world have made large investments in genomics in recent years, which have aided in the development of novel genome editing technologies. Furthermore, the availability of government financing has allowed academic and government institutes to conduct extensive genome editing/engineering research. For instance, in March 2020, Genome Canada received US$ 15 million from the Ministry of Innovation, Science, and Industry (Science) to support 11 genomic research initiatives in the health, agricultural, and environment sectors. Provincial governments, industries, and research partners will contribute a total of US$ 29.7 million to these research projects. The projects involve ovarian and cervical cancer research. The number of genomics research initiatives has increased significantly as a result of major government investments in this sector boosting the genome editing technologies market's growth over the forecast period.

The rise in the incidence of cancer and infectious diseases

Cancer incidence rates are predicted to rise from 20 million new cases per year in 2020 to more than 30 million new cases per year by 2040. Genome editing technologies provide new opportunities in fundamental cancer research and diagnostics, with advantages such as simple design, rapid operation, low cost, and robust scaling, introducing CRISPR/Cas as a rapidly evolving editing technique that is applicable to almost all genomic targets. Several genome editing techniques, including zinc finger endonuclease (ZFN), transcription activator-like effector nuclease (TALEN), and the clustered regularly interspaced short palindromic repeats/CRISPR associated nuclease (CRISPR/Cas) system, have been developed to provide efficient gene editing for the treatment of cancers, infectious diseases, and genetic disorders

Where are the Market Opportunities?

CRISPR Cas9 Technology to widen its applicationCRISPR-Cas9 is one of the most significant discoveries of the twenty-first century. Since its inception in 2012, this gene-editing technology has transformed biology research, making illness research easier and medication discovery faster. The technique is also having a substantial influence on crop development, food production, and industrial fermentation operations. CRISPR-Cas9 technology has huge potential in the pharmaceutical business. Scientists are tackling CRISPR-Cas technology, testing its possibilities and limits as a medical tool. It is being tested for treating diseases in humans such as cancer, blood disorders, blindness, AIDS, and genetic disorder such as Cystic fibrosis, hemophilia, -thalassemia, Alzheimer's, Huntington's, Parkinson's, tyrosinemia, Duchenne muscular dystrophy, Tay-Sachs, and fragile X syndrome disorders.

Competitive LandscapeThe major players operating in the Genome Editing Technologies market are Thermo Fisher Scientific Inc., Merck KGaA, GenScript, Sangamo Therapeutics Inc., Lonza, Editas Medicine, CRISPR Therapeutics AG, Agilent Technologies Inc., Precision Biosciences, and Tecan Life Sciences. These major players operating in this market have adopted various strategies comprising M&A, investment in R&D, collaborations, partnerships, regional business expansion, and new product launch.

Recent Developments

In April 2022, Thermo Fisher Scientific introduced the new GMP-manufactured Gibco CTS TrueCut Cas9 Protein. TrueCut Cas9 proteins are manufactured with United States Pharmacopeia standards in mind, including traceability documentation, aseptic manufacturing, and safety testing.

In February 2022, CRISPR Therapeutics, a biopharmaceutical company focused on developing transformative gene-based medicines for serious diseases, and ViaCyte, Inc., a clinical-stage regenerative medicine company developing novel cell replacement therapies have collaborated to address diseases with significant unmet needs, announced the first patient has been dosed in the Phase 1 clinical trial of VCTX210 for the treatment of type 1 diabetes (T1D)..

To find more Visiongain research reports on the Pharma sector, click on the following links:

Do you have any custom requirements we can help you with?Any need for a specific country, geo region, market segment or specific company information? Contact us today, we can discuss your needs and see how we can help:dev.visavadia@visiongain.com

About Visiongain

Visiongain is one of the fastest-growing and most innovative independent market intelligence providers around, the company publishes hundreds of market research reports which it adds to its extensive portfolio each year. These reports offer in-depth analysis across 18 industries worldwide. The reports, which cover 10-year forecasts, are hundreds of pages long, with in-depth market analysis and valuable competitive intelligence data. Visiongain works across a range of vertical markets with a lot of synergies. These markets include automotive, aviation, chemicals, cyber, defence, energy, food & drink, materials, packaging, pharmaceutical and utilities sectors. Our customised and syndicatedmarket research reportsoffer a bespoke piece of market intelligence customised to your very own business needs.

Contact:Dev VisavadiaPR at Visiongain Reports LimitedTel: + 44 0207 336 6100Email:dev.visavadia@visiongain.com

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I Got Critiqued by YouTuber Gutsick Gibbon – Discovery Institute

Posted: August 5, 2022 at 2:20 am

Photo credit: Julielangford, CC BY-SA 3.0 , via Wikimedia Commons.

Earlier this year, a popular evolution YouTuber, Gutsick Gibbon, or Erika, createda video responseto my post here atEvolution News, Do Statistics Prove Common Ancestry? I had reviewed a paper by Baum et al. (2016), Statistical evidence for common ancestry: Application to primates, and how it presents a flawed and weak argument for separate ancestry that ignores the possibility of common design.

Erika is currently pursuing her Masters of Research in Primate Biology, Behavior and Conservation and is the creator of hundreds of punchy, entertaining YouTube videos. Her channels primary focus seems to be debunking Darwin-skeptics. Unfortunately, she does not seem to apply an equally critical eye to evolutionary theory.

While Erika confidently affirms the conclusions of Baum et al. (2016) in multiple videos here,here, andhere her responses do not negate the arguments raised in my initial post.

Before going further I want to remind you that intelligent design (ID) is compatible with both common ancestry and non-common ancestry views. Some of my colleagues here at Discovery Institute support common ancestry while others (like myself) are more skeptical. Thats OK! We all agree that there is evidence for design in nature. Some of us skeptics are interested in exploring potential models where ID and non-common ancestry histories of life intersect. Design does not rise or fall with these models, but they are interesting questions to explore.

Erikasfirst critiquecan be summarized as a complete misunderstanding of ID proponents objection to the paper. We will deal with that in a post tomorrow.

Hersecond critiqueis that ID proponents shouldnt expect others to test their models, but should test the models themselves. Anyone is welcome to test ID concepts if they like, but I dont think that ID proponents were expecting Baum et al. (2016) to test the hypothesis of separate ancestry. Rather, the paper carried out the normal scientific process where one group of scientists tests another groups scientific hypothesis independently. Only, in this case they tested a hypothesis no one supports more on that later. Perhaps most important, ID proponents are involved in testing models of separate ancestry and the exampleherewas provided in the original post.

Herthird critiqueresponded to my key point there are two known mechanisms (design and ancestry) that can produce genetic similarity. Therefore, genetic similarity should not always be used to provide exclusive support for ancestral relatedness when other explanations are possible.

To elaborate on her third critique, Erika argues that there is no genetic demarcation or separation that would mark a stopping place for comparison between species and higher orders of phyla. She is clearly ignoring reproductive barriers here. While I dont think this argument addresses my bolded point above, I am quite curious what she imagines this stopping point would look like if in fact separate ancestry were correct? I speculate that in such cases people expect the only evidence for a discontinuity in biological relatedness would be a vastly different genetic code for each organism or species. This seems to me a false expectation, because human technology shows that even separately designed structures can have deep similarities that go down to their very blueprints or encoding information. Given that, a design hypothesis would lead us to expect functional similarities. I would also say that there are reasons that a good design would make use of a highly similar genetic code for all organisms.

In this part of her argument, Erika also discusses Last Thursdayism. She says because a seemingly hierarchical ancestral pattern exists, if separate ancestry were correct, the designer must be deceptive to leave us such a pattern. In case you arent familiar with Last Thursdayism, it is a concept that a creator or God could make things look a certain way (billions of years old for example) even if he had created everything last Thursday. While I agree there are problems with Last Thursdayism, Last Thursdayism isnt relevant in this case. There are straightforward reasons to expect some degree of tree-like patterns even in a non-common-ancestry-related dataset.

If the seemingly deceptive pattern exists for a functional reason and has a good design explanation, then there really isnt a deceptive pattern. The deceptive pattern is imposed only by materialist lenses and a poor understanding of functional reasons for the similarities.

To summarize the problems with her third critique,as emphasized in myoriginal post, we know and observe two mechanisms that can result in genetic similarity.Design is one (think genetic engineering) and ancestry (think reproduction) the other.Becausetwoknown mechanisms exist to produce genetic similarity, that means,in and of itself, that genetic similarity does not provide evidence for ancestral relatedness. Certain patterns of genetic similarity may do this, but a design pattern, which isnt randomness, was not considered in the Baum et al. (2016) paper and isnt being considered by many in the academic community. Thats what ID proponents are trying to change.

Erikasfourth critiqueis that Winston Ewerts dependency graph(Ewert 2018)is not an actual model of separate ancestry. Winstons central thesis is that the nested hierarchical pattern observed in subsets of genes is better accounted for by a dependency graph. Erika acknowledges this is outside of her field, but she quotes Joshua Swamidass to dismiss it as a model. Ill talk more about her specific points in a later post.

Finally, Erikaslast pointis to address my argument that Baum et al. (2016) cherry picked which genes they would use when constructing their phylogeny: they only used genes they claimed were phylogenetically informative, which could imply a stacked deck. She really did not address my argument and instead made a comment about orphan genes.

I did not feel that Erika provided evidence for how (experimental) or why (conceptual) common design could not result in genetic similarities between species. Instead, there is evidence of design-dependent genetic similarity exploding all around me. I see it in the artificial selection for dogs (breeding for specific traits). I performed it myself in the lab using recombinant DNA technology. And I see it being dreamed about for the future as bringing about incredible advances in human health using CRISPR-Cas9. These are all proof-of-principle examples that design can and does produce genetic similarity in different organisms. Because this mechanism is well established, when we observe genetic similarity, we cant refuse to include design in the conversation.

In my next post I will explain why I think Gutsick was confused about the objection I raised previously to the separate ancestry model in the Baum et al. (2016) paper and attempt to explain the ID position more clearly.

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CRISPR Technology in the Agricultural Industry: Patent and Regulatory Updates – JD Supra

Posted: August 5, 2022 at 2:20 am

Introduction

The ability to edit eukaryotic DNA entails an almost limitless ability to alter the genetic makeup of the plants that become our food. Recently, scientific attention has been directed to applying a class of new gene-editing techniques that utilize CRISPR to food crops for the introduction of commercially desirable traits. Gene-edited crops can have a positive impact on food productivity, quality, and environmental sustainability, and CRISPR is unique in its relative simplicity, robust flexibility, cost-effectiveness, and wide scope of use. The increased use of CRISPR in agriculture has endless applications, the consequences of which are only recently being analyzed.

CRISPR & the Power of Gene Editing

The term CRISPR refers generally to a class of gene-editing mechanisms derived from prokaryotic immune systems. These mechanisms feature two main components: guiding RNA molecules that direct the second component, CRISPR-associated ("Cas") proteins, to the target region of cellular DNA. These Cas proteins induce a double-stranded break in the DNA and allow for targeted manipulation of the desired genetic code. There is incredible diversity in the CRISPR-Cas system and a multitude of different Cas proteins that can be fine-tuned to induce desired changes with high specificityincluding the activation or deactivation of individual genes, or the insertion of genes from other organisms into the target genome.

CRISPR's flexibility stands in sharp contrast to the previous generation of gene-editing technologies, such as Zinc Finger Nucleases and Transcription Activator-Like Effector Nucleases ("TALENs"), which require massive amounts of preemptive research and development and have a far more limited scope of use. This simultaneous precision and flexibility therefore provides ample opportunity for gene-edited optimization of food crops and has already been used in some instances to create, for example, browning-resistant mushrooms. In late 2021, in Japan, the first CRISPR-edited food product was introduced to the global market: tomatoes with high levels of GABA, a naturally occurring neurotransmitter, due to a CRISPR-inactivated gene.

The power of CRISPR has incredible potential for innovation, but the rights and regulations associated with CRISPR have been elusive and, at times, contentious. CRISPR's game-changing technology was the subject of a series of patent priority, inventorship, and, hence, ownership disputes between high-profile research institutionsthe recent results of which have significant implications for global food supplies.

Patent Landscape

Like most cutting-edge technologies, the invention of CRISPR was accompanied by a flurry of patent application filings in the United States and elsewhere, as researchers who brought CRISPR to light sought to protect and monetize their rights as inventors. Numerous academic institutionsincluding Harvard's and MIT's Broad Institute, the University of California, University of Vienna, Vilnius University, The Rockefeller University, and companies such as ToolGen, Inc., Sigma-Aldrich (Millipore Sigma), Caribou Biosciences, Inc., Editas Medicine, Inc., Keygene N.V., Depixus, Blueallele Corp., and CRISPR Therapeutics AG, among numerous other institutions and companieshave secured U.S. and foreign patent rights related to the applications of CRISPR technology. As CRISPR continues to expand in use, especially in the case of CRISPR-edited agriculture that evade many regulations other GMO foods cannot, the complexity of the patent landscape will almost certainly continue to grow.

EU Regulatory Landscape

In general, the EU subjects agricultural products edited with CRISPR technology to the full suite of genetically modified organism ("GMO") premarket approval, safety, and labeling requirements. The primary EU regulation on point, Directive 2001/18/EC (the "GMO Directive"), was promulgated in 2001 by the European Parliament and Council of the European Union. The GMO Directive requires all EU Member States to create appropriate precautionary measures regarding the release of GMOs in the market. However, the definition of GMO in the GMO Directive apparently excludes CRISPR modification, stating that a GMO is as "an organism, with the exception of human beings, in which the genetic material has been altered in a way that does not occur naturally by mating and/or natural recombination."

It was not until 2018 that the EU addressed this gap in the GMO Directive. In July 2018, the Court of Justice of the European Union explained in Case C-528/16 that organisms obtained by mutagenesis are GMOs within the meaning of the GMO Directive. "Only organisms obtained by means of techniques/methods of mutagenesis which have conventionally been used in a number of applications and have a long safety record are excluded from the scope of that directive."

The following year, in November 2019, the Council of the EU formally requested that the European Commission "submit a study in light of the Court of Justice's judgment in Case C-528/16 regarding the status of novel genomic techniques under Union law, and a proposal, if appropriate in view of the outcomes of the study." The 117-page study was issued in April 2021, and ultimately affirms the holding in Case C-528/16, stating that the "study makes it clear that organisms obtained through new genomic techniques [including CRISPR] are subject to the GMO legislation." Based on the study's findings, the European Commission requested public input on proposed legislation for "plants obtained by targeted mutagenesis and cisgenesis and for their food and feed products." The public consultation period expired on July 22, 2022. The European Commission plans to finalize the proposed framework in 2023.

United States Regulatory Landscape

In contrast to the EU approach, the United States does not currently regulate CRISPR-edited agricultural products as GMOs. The United States regulates biotechnology and genetic modification in food through a "Coordinated Framework" between the U.S. Department of Agriculture ("USDA"), Food and Drug Administration ("FDA"), and Environmental Protection Agency ("EPA").

At a high level, the USDA regulates the use of biotechnology in plant products through the Plant Protection Act. The USDA explains that the Plant Protection Act provides the USDA's Animal and Plant Health Inspection Service ("APHIS") with authority to regulate "organisms and products that are known or suspected to be plant pests or to pose a plant pest risk, including those that have been altered or produced through genetic engineering." Further, in 2018, the USDA's Agricultural Marketing Service promulgated the National Bioengineered Food Disclosure Standard, 7 CFR Part 66 (the "BE Disclosure Standard"), which created a "new national mandatory bioengineered [] food disclosure standard" and associated recordkeeping requirements, effective January 1, 2022. The BE Disclosure Standard defines bioengineered food as food products that contain "genetic material that has been modified through in vitro [DNA]" and "for which the modification could not otherwise be obtained through conventional breeding or found in nature." Notably, the USDA has not explicitly clarified whether CRISPR-edited agricultural products are considered "bioengineered foods" and subject to the BE Disclosure Standard. Rather, in a presentation from 2020, the USDA stated that it "intends to make determinations about whether a specific modifications would be considered 'found in nature' or obtained through 'conventional breeding' on a case-by-case basis." (For more information on the BE Disclosure Standard, refer to Jones Day's May 2022 publication, Are Your Labels Up to Date? Assuring Compliance with the USDA's National Bioengineered Food Disclosure Standard.)

Additionally, the FDA regulates the use of biotechnology in plants with a focus on ensuring that foods are safe for human consumption. In 1992, the FDA issued a Statement of Policy regarding Foods Derived from New Plant Varieties, in which the FDA stated that "[t]he regulatory status of a food, irrespective of the method by which it is developed, is dependent upon objective characteristics of the food and the intended use of the food (or its components)." Since then, the FDA has reviewed genetic modifications to food in the context of food additives, such that FDA approval is required to use food additives unless it is generally recognized as safe ("GRAS"). In the opinion of the FDA, a GMO is not GRAS if the altered substance "differs significantly in structure, function or composition from substances found currently in food." In contrast, a GMO is GRAS if it is "naturally occurring" in the food product, even if is bioengineered to be present at a "greater level" than found in nature or if there are "minor variations in molecular structure that do not affect safety." As explained in the introduction, CRISPR technology differs from conventional gene editing because it does not introduce new substances into a product that are not naturally present. Accordingly, CRISPR-edited agricultural products are not generally regulated by the FDA as food additives.

The EPA also reviews the use of biotechnology in plants, as it regulates the distribution, sale, and use of pesticides to ensure that they will "not pose unreasonable risks to human health or the environment when used according to label directions." Further, when the EPA evaluates plant-incorporated protectants ("PIPs"), which are genetically engineered pesticides, the EPA "requires extensive studies containing numerous factors, such as risks to human health, nontarget organisms, and the environment; potential for gene flow; and the need for insect resistance management plans." As such, CRISPR-edited pesticides may be regulated by the EPA as PIPs.

Conclusion

The patent and regulatory landscapes of the use of CRISPR technology in food are continuing to unfold across the world. Accordingly, agriculture companies and the broader agricultural industry should pay close attention to all developments.

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They’re From Argentina, And They’re One Of The Few Labs That’s Been Able To Eliminate THC From Cannabis With Gene Editing. – Nation World News

Posted: August 5, 2022 at 2:20 am

three Entrepreneurs got the tool to remove them cannabis his main psychoactive ingredientThe tetrahydrocannabinol (THC), and managed to figure it out start up among first laboratory In the world to obtain genetically edited variants capable of applying this technology to this crop that could avoid millionaire losses in an industry that goes beyond pharmaceutical use.

Two Biotechnologists and an Economist Tool managed to design reproductive engineering It acts like a biological scissor inspired by an Antarctic bacterium, modifying the genome of cannabis and allow to obtain better plants for medicinal and industrial applications.

We are the first Argentine laboratory that managed to efficiently edit the cannabis genome at an experimental level, with reporter proteins that allow us to monitor the efficiency of the process, he says. Ramiro OliveiraAnimal Biotechnology Expert and CEO biotech chalisa, Created and incubated by company scientists National University of San Martin (that).

stick up for Stephen Hernandoexperts in plant biotechnology, and Alexander Germe, Director of Finance and Project Strategy. The Biotechnological Research Institute of Unsam is located on the Miguelet campus start up Account from last year with authorization from coniset And this national health ministry For your research and development projects with the cannabis plant.

In Argentina, according to Oliveira, they had not yet started applying. biotechnology equipment for the improvement of this plant. The cannabis revolution in the world through biotechnology. It is an industry that is growing rapidly and prohibitionism meant that this crop was not studied scientifically in the world: sooner or later, it will be like another crop of agricultural value, the researchers explain. . We understood this and decided to become a leader in the development of sophisticated tools that allow us to solve problems that are already representative in the industry today. For example, high levels of THC This is causing huge losses to the industrial cannabis growers.

By regulation, the THC level of the cannabis plant cannabis sativa For the manufacture of products for medicinal or pharmaceutical use it cannot exceed 0.3%. Above that value, it is considered a crop with psychoactive properties. The plant naturally expresses a higher percentage of THC than is allowed and must be subjected to complex extraction processes to eliminate this cannabinoid with a high cost.

These genetically edited crops will be the solution to the problem that generates millionaire losers for the industry and will position us as a benchmark in biotechnology applied to this crop, says Oliveira.

With previous experience in highly complex biotechnology projects, such as the cloning of horses and improvements with genetic engineering of crops such as soybeans, wheat and alfalfa, the team has since last year focused on their new venture, the main objective of which is to : Developing them based on technology known as own gene editing tools CRISPR-Cas9 To be able to optimize the properties of the plant. with them biological scissors, the researchers claim they can edit DNA precisely and efficiently.

The first step was to identify the gene that needed to be worked on. In this case, it is associated with the presence of THC in the plant. The team then designed biological scissors to introduce into the plant: an enzyme that cuts d n With a ribonucleic acid guide (arn) to accurately orient the region of the genome to modify it.

We apply an editing strategy in which genetic material from outside the plant is not used. For that we separate the cells from the plants and remove their cell wall. These cells (protoplasts) co-incubate with enzymes and guide [de ARN] To carry out editing, which aims to disrupt and lose its function as the gene responsible for THC synthesis. This makes it possible to produce better plants, without being considered transgenic, explains Oliveira. Country, Once these edited cells are obtained, they are selected and reproduced in a new plant through in vitro culture [en pequeos recipientes con agar rico en hormonas y nutrientes], In the greenhouse, they are molecularly evaluated to check their growth success.

Laboratory tests showed that they were able to fine-tune that process by effectively modifying genome From the cannabis plant. This confirms that our technology works and is a great step forward in science and technology for Argentina. It is a great progress both in the country and in the region that will allow us to think about improvements in other developments productive interest for this harvest, highlights Oliveira.

The team argues that the tool has no limits, as it also allows you to boost the level of others. cannabinoid Plants that are expressed in low concentrations but which have high healing potential and have not yet been studied due to difficulties in purifying them in quantities. It also makes it possible to regulate the ratio of different cannabinoids to their use. medicine or cosmeticsAs medical research advances in defining dosage limits.

Points to development of another application that the team oversees industrial hemp, It is anticipated that hemp fiber will compete with cotton in a few years, predict the entrepreneurs, who are seeing potential demand from other industries in the world. In that case, the cannabis industry will demand better plants that can be applied to comprehensive management. The tools we develop serve to produce plants that are more resistant to inclement weather and pathogens. With these technologies, we will be able to develop non-transgenic plants that are more productive, efficient in use of resources and with lower production costs.

Last month, ahead of a presentation by the team, National Commission on Agricultural Technology (Konabia) informed them that the development of THC-free plant varieties with CRISPR-Cas9 technology is not considered transgenic. This means that these plants can be on the market quickly in two or three years. This not only guarantees the safety of this type of crop, but also saves the company between 50 and 100 million dollars, which It will be necessary to regulate and market a crop considered to be transgenic, concludes the team.

For this project, the team received financial support from ministry of producer development And this Under Secretary of the Knowledge Economy,

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Novavax Announces Initiation of Phase 2b/3 Hummingbird Global Clinical Trial for the Novavax COVID-19 Vaccine in Children Aged Six Months Through 11…

Posted: August 5, 2022 at 2:20 am

GAITHERSBURG, Md., Aug. 4, 2022 /PRNewswire/ -- Novavax,Inc. (Nasdaq: NVAX), a biotechnology company dedicated to developing and commercializing next-generation vaccines for serious infectious diseases, today announced the initiation of its Phase 2b/3 Hummingbird global clinical trial. The trial will evaluate the safety, effectiveness (immunogenicity), and efficacy of two doses of the Novavax COVID-19 vaccine (NVX-CoV2373) in younger children aged six months through 11 years, followed by a booster at six months after the primary vaccination series.

"We are excited to begin the Hummingbird trial to study Nuvaxovid's efficacy in children as young as six months through age 11," said Stanley C. Erck, President and Chief Executive Officer, Novavax. "With a successful trial, we may have the opportunity to offer our COVID-19 vaccine to all age groups aged six months and older for protection against this ongoing pandemic."

The trial will assess the Novavax COVID-19 vaccine in infants (six through 23 months of age), toddlers (two through five years) and children (six through 11 years). The trial is an age de-escalation trial and age groups will be tested sequentially. Participants have begun dosing in the six to 11-year-old age group. The trial will also have sentinel cohorts in each age group and cohort progression and age-de-escalation will occur after safety review.

The trial will seek to enroll 3,600 participants in the US, Mexico, Colombia, Argentina, Spain, UK, South Africa, Philippines, and Brazil. Initial results are expected in Q1 2023.

About the Novavax COVID-19 vaccine (NVX-CoV2373)

The Novavax COVID-19 vaccine (NVX-CoV2373) is a protein-based vaccine engineered from the genetic sequence of the first strain of SARS-CoV-2, the virus that causes COVID-19 disease. The vaccine was created using Novavax' recombinant nanoparticle technology to generate antigen derived from the coronavirus spike (S) protein and is formulated with Novavax' patented saponin-based Matrix-M adjuvant to enhance the immune response and stimulate high levels of neutralizing antibodies. The Novavax COVID-19 vaccine contains purified protein antigen and can neither replicate, nor can it cause COVID-19.

The Novavax COVID-19 vaccine is packaged as a ready-to-use liquid formulation in a vial containing ten doses. The vaccination regimen calls for two 0.5 ml doses (5 mcg antigen and 50 mcg Matrix-M adjuvant) given intramuscularly 21 days apart. The vaccine is stored at 2- 8 Celsius, enabling the use of existing vaccine supply and cold chain channels. Use of the vaccine should be in accordance with official recommendations.

Novavax has established partnerships for the manufacture, commercialization and distribution of its COVID-19 vaccine worldwide. Existing authorizations leverage Novavax' manufacturing partnership with Serum Institute of India, the world's largest vaccine manufacturer by volume. They will later be supplemented with data from additional manufacturing sites throughout Novavax' global supply chain.

About Matrix-M Adjuvant

Novavax' patented saponin-based Matrix-M adjuvant has demonstrated a potent and well-tolerated effect by stimulating the entry of antigen-presenting cells into the injection site and enhancing antigen presentation in local lymph nodes, boosting immune response.

About Novavax

Novavax, Inc. (Nasdaq: NVAX) is a biotechnology company that promotes improved health globally through the discovery, development, and commercialization of innovative vaccines to prevent serious infectious diseases. The company's proprietary recombinant technology platform harnesses the power and speed of genetic engineering to efficiently produce highly immunogenic nanoparticles designed to address urgent global health needs. The Novavax COVID-19 vaccine, has received authorization from multiple regulatory authorities globally, including the U.S., EC and the WHO. The vaccine is currently under review by multiple regulatory agencies worldwide, including for additional indications and populations such as adolescents and as a booster. In addition to its COVID-19 vaccine, Novavax is also currently evaluating a COVID-seasonal influenza combination vaccine candidate in a Phase 1/2 clinical trial, which combines NVX-CoV2373 and NanoFlu*, its quadrivalent influenza investigational vaccine candidate, and is also evaluating an Omicron strain-based vaccine (NVX-CoV2515) as well as a bivalent Omicron-based / original strain-based vaccine. These vaccine candidates incorporate Novavax' proprietary saponin-based Matrix-M adjuvant to enhance the immune response and stimulate high levels of neutralizing antibodies.

For more information, visitwww.novavax.comand connect with us on LinkedIn.

*NanoFlu identifies a recombinant hemagglutinin (HA) protein nanoparticle influenza vaccine candidate produced by Novavax. This investigational candidate was evaluated during a controlled phase 3 trial conducted during the 2019-2020 influenza season.

Forward-Looking Statements

Statements herein relating to the future of Novavax, its operating plans and prospects, its partnerships, the timing of clinical trial results, the ongoing development of NVX-CoV2373, including an Omicron strain based vaccine and bivalent Omicron-based / original strain based vaccine, a COVID-seasonal influenza investigational combination vaccine candidate, the scope, timing and outcome of future regulatory filings and actions, including Novavax' plans to supplement existing authorizations with data from the additional manufacturing sites in Novavax' global supply chain, additional worldwide authorizations of NVX-CoV2373 for use in adults and adolescents, and as a booster, the evolving COVID-19 pandemic, the potential impact and reach of Novavax and NVX-CoV2373 in addressing vaccine access, controlling the pandemic and protecting populations, the efficacy, safety and intended utilization of NVX-CoV2373, and the expected administration of NVX-CoV2373 are forward-looking statements. Novavax cautions that these forward-looking statements are subject to numerous risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. These risks and uncertainties include, without limitation, challenges satisfying, alone or together with partners, various safety, efficacy, and product characterization requirements, including those related to process qualification and assay validation, necessary to satisfy applicable regulatory authorities; difficulty obtaining scarce raw materials and supplies; resource constraints, including human capital and manufacturing capacity, on the ability of Novavax to pursue planned regulatory pathways; unanticipated challenges or delays in conducting clinical trials; challenges meeting contractual requirements under agreements with multiple commercial, governmental, and other entities; and those other risk factors identified in the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of Novavax' Annual Report on Form 10-K for the year ended December 31, 2021 and subsequent Quarterly Reports on Form 10-Q, as filed with the Securities and Exchange Commission (SEC). We caution investors not to place considerable reliance on forward-looking statements contained in this press release. You are encouraged to read our filings with the SEC, available at http://www.sec.govand http://www.novavax.com, for a discussion of these and other risks and uncertainties. The forward-looking statements in this press release speak only as of the date of this document, and we undertake no obligation to update or revise any of the statements. Our business is subject to substantial risks and uncertainties, including those referenced above. Investors, potential investors, and others should give careful consideration to these risks and uncertainties.

Contacts:

InvestorsErika Schultz | 240-268-2022[emailprotected]

MediaAli Chartan or Giovanna Chandler | 202-709-5563[emailprotected]

SOURCE Novavax, Inc.

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Novavax Announces Initiation of Phase 2b/3 Hummingbird Global Clinical Trial for the Novavax COVID-19 Vaccine in Children Aged Six Months Through 11...

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