Harvard University and National Resilience, Inc. (Resilience), a manufacturing and technology company, have established a five-year R&D alliance with a $30 million commitment from Resilience directed toward the development of complex medicines, including biologics, vaccines, nucleic acids, and cell and gene therapies.

Under the alliance agreement coordinated by Harvards Office of Technology Development (OTD), Resilience will fund faculty-initiated research focused on certain novel therapeutic and biomanufacturing technologies pioneered in University labs. The alliance also anticipates that these Harvard innovations may be commercially advanced by new companies formed by Resilience expressly to drive these technologies into clinical development and commercialization.

An initial technology platform has already been identified for incubation under the alliance, with promising applications in skeletal muscle disorders. In the Harvard lab of Lee Rubin, professor of Stem Cell and Regenerative Biology, researchers have developed a means to culture millions of cells in vitro that behave like skeletal muscle stem cells (satellite cells), retaining their regenerative potential, for use in possible cell therapies. Resilience is now funding the labs continuing work on the platform, aiming to further validate it, in a project led by staff scientist Feodor Price.

Meanwhile, Resilience has formed an entity called Circle Therapeutics, anticipating that Circle may carry the technology forward under license.

For six decades since the discovery of the satellite cell, it has not been possible to expand therapeutic numbers of satellite cells in vitro, until we made real headway on it at Harvard, said Rubin. Were truly excited for the possible therapeutic impact of our innovations.

Our mission at Resilience is to make a new generation of complex medicines, such as curative gene therapies, life-saving vaccines and immune-system-boosting cell therapies, more accessible to people in need, said Rahul Singhvi, chief executive officer of Resilience. Current biomanufacturing processes pose significant hurdles to making these medicines quickly, and at scale, which is why we are excited to work with researchers at Harvard to identify and develop the technologies needed to make this future a reality.

The Rubin Labs platform to expand and maintain in vitro-derived satellite cells could lead to transformative cell therapies, said Vivian Berlin, executive director, HMS, at Harvard OTD, who leads OTDs Corporate Alliances team. With prior support from the Blavatnik Biomedical Accelerator, the team has compellingly demonstrated the clinical relevance of this work. Now with Resiliences focused funding and experience in the development of complex medicines, we hope to set it on a clear path toward benefiting patients.

Going forward, Resilience and Harvard will jointly issue a call for proposals to identify additional research projects to be funded at Harvard. Under the terms of the alliance, Resilience will receive an option to license technologies arising from funded projects.

This research alliance with Resilience will help support biomedical innovation at Harvard, said Isaac Kohlberg, Harvards chief technology development officer and senior associate provost. Collaborating to both advance Harvard science and place arising technologies with dedicated new ventures, we can provide yet another valuable source of support and industry expertise to translational biomedical researchers across Harvards Schools as they seek to impact human health for the better.

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Harvard's R&D alliance with Resilience to advance manufacture of complex medicines - Harvard Gazette

Published: Oct. 15, 2021 at 8:50 AM EDT|Updated: 18 hours ago

HANGZHOU, China, Oct. 15, 2021 /PRNewswire/ -- On Oct. 11th, 2021, CellOrigin Inc. released data about its second generation of iPSC-CAR-Macrophage which has a genetically integrated secondary signal to confer controlled CAR-iMac polarization, in the 5th International Conference of IGC China, 2021, Beijing.

Recently, CellOrigin Biotech, a company committed to iPSC-derived innate immune cell therapeutics, has announced a new round of investment by Kunlun Capital. The investment will be used for the CMC development for its current pipeline of iPSC-derived innate immune cells such as iPSC-CAR-Macrophage and rationlly designed iPSC-NK cells. Before, CellOrigin have also acquired investment from Shulan Health and Nest. Bio Ventures.

CellOrigin Biotech has a long term focus on iPSC-derived innate immune cells and its applications in new cancer immune cells. Dr. Jin Zhang, the scientific co-founder of CellOrigin used to be trained as a research fellow at the Boston Children's Hospital and Harvard Medical School. Now, his team worked closely with clinicians at the First Affiliated Hospital of Zhejiang University, and for the first time his team reported the induced pluripotent stem cell or iPSC-derived CAR-macrophages (CAR-iMac), and its applications in cancer immunotherapies.

CellOrigin Biotech holds the domestic and global patents for iPSC-derived CAR-Macrophage, and the engineering for polarization. With this proprietary platform, they are collaborating with research groups in genome engineering and synthetic biology worldwide to fully unleash the potential of iPSC-derived immune cells, which are highly editable, expandable and clonal. Eventually, they would like to achieve a goal of bring more effective, universal and safe immune cell products to cancer patients, especially for those with solid tumors. The investigator initiated trials has been initiated at the First Hospital of Zhejiang University. The core proprietary technology platform and the core patents including the engineered macrophages from pluripotent stem cells has been authorized and is in the process of entering different countries worldwide.

To support the CMC of its pipeline products, on Oct 1st, CellOriginhas announced the launch of its 3000 square feet GMP facility at Hangzhou, China.

About Kunlun Capital

Founded in 2015, Kunlun capital is committed to long-term value investment, establishing long-term partnership with entrepreneurs, and focusing on investing in enterprises with high technical barriers, excellent founding team and explosive growth potential. In recent years, Kunlun capital has successively invested in KEYA Medical, EdiGene, Cytek (NASDAQ:CTKB), Hui-Gene Therapeutics, OBiO, Okeanos, Ucell Biotech, CellOrigin, Soonsolid, Inke (HK:03700), Dada (NASDAQ:DADA), Dreame, Bamboocloud, Pony.ai, PingCAP, Leyan Technologies.

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SOURCE Cell Origin

The above press release was provided courtesy of PRNewswire. The views, opinions and statements in the press release are not endorsed by Gray Media Group nor do they necessarily state or reflect those of Gray Media Group, Inc.

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CellOrigin secured a new round of investment for developing its globally proprietary iPSC-CAR-Macrophage technology platform - WWNY

TEL AVIV, Israel, Oct. 13, 2021 /PRNewswire/ --BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a late clinical-stage biopharmaceutical company focused on oncology, today announced positive results from a pharmacoeconomic study evaluating the cost-effectiveness of using investigational drug Motixafortide as a primary stem cell mobilization (SCM) agent on top of granulocyte colony stimulating factor (G-CSF), versus G-CSF alone, in multiple myeloma patients undergoing autologous stem cell transplantation (ASCT). The study was performed by the Global Health Economics and Outcomes Research (HEOR) team of IQVIA, and was a pre-planned study conducted in parallel with the GENESIS Phase 3 trial. These results, together with the highly significant and clinically meaningful data from the GENESIS trial, strongly support the potential use of Motixafortide, on top of G-CSF, as the standard of care in SCM for ASCT.

The study concluded that the addition of Motixafortide to G-CSF (the current standard of care) is associated with a statistically significant decrease in health resource utilization (HRU) during the ASCT process, compared to G-CSF alone. Based on the significantly higher number of mobilized cells and the lower number of apheresis sessions, lifetime estimates show quality-adjusted-life-year (QALY) benefits and net cost savings of ~$17,000 (not including the cost of Motixafortide), versus G-CSF alone. The study findings, combined with model estimates, suggest that the use of Motixafortide, on top of G-CSF, as the standard of care in mobilization for ASCT, could be a cost-effective option in the US, based on accepted willingness-to-pay (WTP) values for healthcare payers.

"The compelling cost savings identified by this rigorously designed study strongly support the Company's view that Motixafortide, in combination with G-CSF, can become the new standard of care as an upfront, or primary, therapy for all multiple myeloma patients undergoing autologous stem cell transplantation," stated Philip Serlin, Chief Executive Officer of BioLineRx. "Based on data from the GENESIS trial showing that nearly 90% of patients collected an optimal number of cells for transplantation following a single administration of Motixafortide and in only one apheresis session, versus less than 10% for G-CSF alone, the pharmacoeconomic study demonstrates that use of Motixafortide on top of G-CSF can save $17,000 per patient, not including the cost of Motixafortide. These cost savings should leave substantial room in the future to optimize our pricing strategy for Motixafortide at product launch and thereafter, if approved.

"It is also important to note that fewer administrations and apheresis sessions confer meaningful safety and time benefits to patients. In addition, the significantly higher median number of cells collected in one apheresis session ~11 million using Motixafortide on top of G-CSF versus ~2 million for G-CSF alone not only enables transplantation of an optimal number of cells, with the potential to significantly save on time to engraftment, it also permits the retention of enough cells for cryopreservation in the event that an additional transplantation is required in the future. Lastly, higher levels of certainty regarding the number of apheresis sessions required for mobilization could enable more efficient utilization of apheresis units at transplantation institutions, where there is often a shortage of available machines.

"We believe the data from the GENESIS study, together with the results from this pharmacoeconomic study, set Motixafortide apart from all other mobilization agents either currently available or in development. If approved, Motixafortide represents a significant advancement in SCM to the benefit of patients and payers alike, and, to that end, we remain on track to submit a New Drug Application (NDA) to the FDA in the first half of next year," Mr. Serlin concluded.

About the Pharmacoeconomic Study

The pharmacoeconomic study analyzed healthcare resource utilization (HRU) observed during the Phase 3 GENESIS trial, which randomized 122 patients into two arms: Motixafortide plus G-CSF (n=80) or placebo plus G-CSF (n=42). HRU data points collected include: (1) the number of Motixafortide and G-CSF doses, as well as the number of apheresis sessions performed, in primary mobilization; (2) the percentage of patients needing rescue mobilization due to poor primary mobilization, including the number of apheresis sessions needed and the number of G-CSF and plerixafor doses required; and (3) hospitalization costs related to conditioning and transplantation, including length of stay. Quality-adjusted life years gained (QALY) from published literature were also incorporated into the model. Motixafortide plus G-CSF was associated with a statistically significant HRU decrease during the autologous stem cell transplantation process compared to standard-of-care G-CSF alone. Given the higher number of mobilized cells and lower number of apheresis sessions, lifetime estimates show quality-adjusted-life-year (QALY) benefits and net cost savings of ~$17,000 (not including the cost of Motixafortide), versus the current standard of care.

About the GENESIS Phase 3 Trial

The GENESIS Phase 3 trial (NCT03246529) was initiated in December 2017. GENESIS was a randomized, placebo-controlled, multicenter study, evaluating the safety, tolerability and efficacy of Motixafortide and G-CSF, compared to placebo and G-CSF, for the mobilization of hematopoietic stem cells for autologous transplantation in multiple myeloma patients. The primary objective of the study was to demonstrate that only one dose of Motixafortide on top of G-CSF is superior to G-CSF alone in the ability to mobilize 6 million CD34+ cells in up to two apheresis sessions. Additional objectives included time to engraftment of neutrophils and platelets and durability of engraftment, as well as other efficacy and safety parameters. The study successfully met all primary and secondary endpoints with an exceptionally high level of statistical significance (p<0.0001), including approximately 90% of patients who mobilized the target number of cells for transplantation with only one administration of Motixafortide and in only one apheresis session.

About Stem Cell Mobilization for Autologous Stem Cell Transplantation

Autologous stem cell transplantation (ASCT) is part of the standard treatment paradigm for a number of blood cancers, including multiple myeloma, non-Hodgkin's lymphoma and other lymphomas. In eligible patients, ASCT is performed after initial (induction) therapy, and, in most cases, requires consecutive-day clinic visits for the mobilization and apheresis (harvesting) phases, and full hospitalization for the conditioning chemotherapy and transplantation phases until engraftment. The associated burden is therefore significant patients experience clinically relevant deteriorations in their quality of life during ASCT, and healthcare resource use throughout the ASCT phases is particularly intense. Therefore, new interventions impacting the ASCT process have the potential for relieving some of the clinical burden for transplanted patients, the logistical burden for the apheresis units, and the financial burden for healthcare providers and payers.

Described simply, ASCT consists of: (1) mobilizing the patient's own stem cells from his/ her bone marrow to the peripheral blood for removing (harvesting) via an apheresis procedure; (2) freezing and storing the harvested cells until they are needed for transplantation; (3) providing a conditioning treatment, such as high-dose chemotherapy or radiation, to kill the remaining cancer cells the day before transplant; and (4) infusing the stored stem cells back to the patient intravenously via a catheter.

To mobilize the patient's stem cells from the bone marrow to the peripheral blood for harvesting, the current standard of care includes the administration of 5-8 daily doses of granulocyte colony stimulating factor (G-CSF), and the performance of 1-4 apheresis sessions. For patients unable to mobilize sufficient numbers of cells for harvesting during this primary mobilization phase, rescue therapy is carried out, consisting of 1-4 doses of plerixafor on top of G-CSF, and the performance of an additional number of apheresis sessions as necessary. In light of this, an agent with superior mobilization activity may significantly reduce the mobilization and harvesting burden and associated risks of the ASCT process and lead to significant clinical and resource benefits.

About BioLineRx

BioLineRx Ltd. (NASDAQ/TASE: BLRX) is a late clinical-stage biopharmaceutical company focused on oncology. The Company's business model is to in-license novel compounds, develop them through clinical stages, and then partner with pharmaceutical companies for further clinical development and/or commercialization.

The Company's lead program, Motixafortide (BL-8040), is a cancer therapy platform that was successfully evaluated in a Phase 3 study in stem cell mobilization for autologous bone-marrow transplantation, as well as reporting positive results from a pre-planned pharmacoeconomic study, and is currently in preparations for an NDA submission. Motixafortide was also successfully evaluated in a Phase 2a study for the treatment of pancreatic cancer in combination with KEYTRUDA and chemotherapy under a clinical trial collaboration agreement with MSD (BioLineRx owns all rights to Motixafortide), and is currently being studied in combination with LIBTAYO and chemotherapy as a first-line PDAC therapy.

BioLineRx is also developing a second oncology program, AGI-134, an immunotherapy treatment for multiple solid tumors that is currently being investigated in a Phase 1/2a study.

For additional information on BioLineRx, please visit the Company's website at http://www.biolinerx.com, where you can review the Company's SEC filings, press releases, announcements and events.

Various statements in this release concerning BioLineRx's future expectations constitute "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include words such as "may," "expects," "anticipates," "believes," and "intends," and describe opinions about future events. These forward-looking statements involve known and unknown risks and uncertainties that may cause the actual results, performance or achievements of BioLineRx to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Factors that could cause BioLineRx's actual results to differ materially from those expressed or implied in such forward-looking statements include, but are not limited to: the initiation, timing, progress and results of BioLineRx's preclinical studies, clinical trials and other therapeutic candidate development efforts; BioLineRx's ability to advance its therapeutic candidates into clinical trials or to successfully complete its preclinical studies or clinical trials; BioLineRx's receipt of regulatory approvals for its therapeutic candidates, and the timing of other regulatory filings and approvals; the clinical development, commercialization and market acceptance of BioLineRx's therapeutic candidates; BioLineRx's ability to establish and maintain corporate collaborations; BioLineRx's ability to integrate new therapeutic candidates and new personnel; the interpretation of the properties and characteristics of BioLineRx's therapeutic candidates and of the results obtained with its therapeutic candidates in preclinical studies or clinical trials; the implementation of BioLineRx's business model and strategic plans for its business and therapeutic candidates; the scope of protection BioLineRx is able to establish and maintain for intellectual property rights covering its therapeutic candidates and its ability to operate its business without infringing the intellectual property rights of others; estimates of BioLineRx's expenses, future revenues, capital requirements and its needs for additional financing; risks related to changes in healthcare laws, rules and regulations in the United States or elsewhere; competitive companies, technologies and BioLineRx's industry; risks related to the COVID-19 pandemic; and statements as to the impact of the political and security situation in Israel on BioLineRx's business. These and other factors are more fully discussed in the "Risk Factors" section of BioLineRx's most recent annual report on Form 20-F filed with the Securities and Exchange Commission on February 23, 2021. In addition, any forward-looking statements represent BioLineRx's views only as of the date of this release and should not be relied upon as representing its views as of any subsequent date. BioLineRx does not assume any obligation to update any forward-looking statements unless required by law.

Contact:Tim McCarthyLifeSci Advisors, LLC+1-212-915-2564[emailprotected]

or

Moran MeirLifeSci Advisors, LLC+972-54-476-4945[emailprotected]

SOURCE BioLineRx Ltd.

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BioLineRx Announces Positive Results from Pharmacoeconomic Study Positioning Motixafortide as Potential Standard of Care in Stem Cell Mobilization -...

Imagine youre about to go on a cross-country trip, stopping at spots along the way to admire local attractions. Youd probably want to have a road atlas handy, containing maps at different scales, covering both the major highways and the roads of smaller cities and towns or at least a GPS that can access a digital atlas with this information.

Until recently, cancer researchers have been like cross-country travelers with only a few maps of a few popular cities. And because of how fast some cancers grow, the maps quickly go out of date. This situation has hindered doctors ability to understand whats really going on inside tumors and develop effective treatments.

The Human Tumor Atlas Network (HTAN) was created to change that. It aims to develop high-resolution maps of many kinds of cancer so that doctors could have a more-complete view of the textured terrain of tumors including how they change over time to become more deadly. HTAN is funded by the National Cancer Institute and involves a consortium of cancer centers across the United States.

After several years of painstaking research, the first such atlas from investigators at Memorial Sloan Kettering Cancer Center for small cell lung cancer is now ready for viewing, and its full of new insights.

The most exciting thing we found is a rare population of stem-like cells within these tumors that is closely correlated with patient outcomes, explains Charles Rudin, a physician-scientist at MSK who co-led the lung cancer project. The more enriched they are in the tumor, the worse the prognosis.

Not only that, but these stem-like cells have metastatic properties meaning theyre prone to spread and researchers found them across many SCLC tumors that otherwise were very different.

That was a massive surprise, says Dana Peer, a computational biologist at MSK who is a principal investigator of the HTAN and co-led the lung cancer atlas project. It raises the possibility that this tiny fraction of cells could be driving metastatic behavior across tumors.

Small cell lung cancer is one of the deadliest cancers. It tends to spread early and aggressively; two-thirds of cases are already metastatic at diagnosis. Chemotherapy is not very effective. The researchers hope their new atlas, which was published October 14, 2021, in the journal Cancer Cell, will lead to improvements in care for people with the disease.

Building the atlas required years of collaborative work from two groups with very different areas of expertise: clinicians like Dr. Rudin with disease-specific expertise in small cell lung cancer and computational biologists like Dr. Peer and her team.

Dr. Rudin points to the fact that there are four co-first authors on the paper an unusual occurrence as evidence of the diversity of skillsets needed to complete a study like this. The co-first authors are Joseph Chan, lvaro Quintanal-Villalonga, Vianne Ran Gao, and Yubin Xie.

Dr. Peer, Chair of the Computational and Systems Biology Program at the Sloan Kettering Institute, took the lead on the computational side of things. She is an expert in single-cell RNA seq (scRNAseq), a technique that allows scientists to get a detailed picture of which genes are turned on in many hundreds of cells at the same time.

By applying scRNAseq to SCLC tumor specimens obtained from patients at MSK, Dr. Peer and her team were able to find this rare population of stem cell-like cells lurking amidst the cells of the surrounding tumor, like locating a needle in a haystack.

We would never have been able spot these cells with bulk sequencing. We really needed single cell analysis to find them.

We would never have been able spot these cells with bulk sequencing, she says. We really needed single cell analysis to find them. (Bulk sequencing is what researchers would do before scRNAseq was available essentially putting the tumor in a blender and sequencing all the RNA that fell out.)

The single cell technique also allowed the team to go further. Within the cells making up this tiny population, one gene stood out: PLCG2. This gene makes a protein that acts as a second messenger it relays signals from one protein to another.

PLCG2 did not initially strike me as the sort of gene that would be involved in regulating stem cell populations, Dr. Rudin says. It seems like more of a worker bee.

But indeed, PLCG2 does seem to be playing an important role. The gene is most highly expressed in this stem cell-like population, the scientists found. And when they experimentally increased or lowered its activity in cancer cell lines, it altered the ability of the cancer cells to metastasize.

They researchers think that these PLCG2-high cells could be part of the explanation for SCLCs aggressiveness. If so, it could open up new possibilities for treatment.

The thought is that if we can develop strategies to selectively target this cell population, we might be able to suppress metastasis and ultimately improve outcomes for patients with small cell lung cancer, Dr. Rudin says.

What we really want to do is try to stop metastasis in its tracks, Dr. Peer adds. But to do that, we need to better understand these rare cell populations that seem to be driving it. Thats the goal of this atlas.

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Molecular Atlas of Small Cell Lung Cancer Reveals Unusual Cell Type That Could Explain Why Its So Aggressive - On Cancer - Memorial Sloan Kettering