On October 13, 2020 EdiGene, Inc., which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, reported the successful completion of a RMB 450 million (approximately USD 67 million) Series B financing (Press release, EdiGene, OCT 13, 2020, View Source [SID1234568346]). 3H Health Investment led the round and other new investors included Sequoia Capital China, Alwin Capital and Kunlun Capital, along with continued support by previous investors, including IDG Capital, Lilly Asia Venture, Huagai Capital and Green Pine Capital Partners. Proceeds from the financing will be used to advance the company’s pipeline into clinics and to expand the team.

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Minchuan Wang, Ph.D., Partner of 3H Health Investment commented: "As one of the most disruptive biomedical technologies, gene editing is rapidly moving toward clinics globally. EdiGene is leading the wave in China, as they have established outstanding research and development capabilities, and more importantly, they have developed an impressive portfolio of proprietary gene editing tools and product candidates. We are pleased to lead the Series B financing, and are excited to join force with EdiGene and other investors to bring life-changing innovative gene-editing therapies to patients in China and globally. We look forward to contributing our clinical, business, and policy resources to further strengthen the company’s capabilities, and are confident that EdiGene is well-positioned to be a leader in gene-editing therapeutics."

"We have raised approximately RMB 700 million (USD 100 million) in the past two years. We are delighted to add these top tier investors in closing of our Series B financing, and are grateful for the continuous support from the current investors," said Dong Wei, Ph.D.，CEO of EdiGene, "The round enables us to further scale up and transform our pipeline into clinical-stage, which is also a big step forward in building a globally competitive gene editing company. More importantly, we are closer to realizing our mission of bringing innovative and high-quality gene-editing therapies to patients in need."

"We are very pleased to have the support and partnership from our investors, which propels the company to an exciting new stage," said Wensheng Wei, Scientific Founder of EdiGene, "Together with the investors, we look forward to translating cutting-edge gene editing technologies into innovative therapies, bringing hope and health to patients and their families."

Founded in 2015, EdiGene has established four gene editing based platforms and is advancing its early stage programs into clinical development for patients with genetic diseases and cancer. The four platforms are ex vivo genome-editing platforms for hematopoietic stem cells and T cells, in vivo therapeutic platform based on RNA base editing, and high-throughput genome-editing screening to discover novel targeted therapies. In addition, EdiGene has launched GMP manufacturing facility in 2018 in Guangdong Province.

On October 12, 2020 Baylor College of Medicine reported that Natural killer T (NKT) cells, a type of immune cells known for their potent anti-cancer properties in murine tumor models, have been developed into a novel form of immunotherapy to treat patients with cancer (Press release, Baylor College of Medicine, OCT 12, 2020, View Source [SID1234568607]).

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Researchers at Baylor College of Medicine and the University of North Carolina at Chapel Hill have genetically modified human NKT cells with a chimeric antigen receptor (CAR) that enables them to specifically recognize and attack neuroblastoma, a form of childhood cancer. Expressed with the CAR is interleukin-15 (IL-15), a natural protein that supports NKT cell survival.

In the study, appearing in Nature Medicine, researchers present interim results from an ongoing clinical trial showing that the modified cells are safe, localize to tumors, and, in one of three patients, induced an objective response with regression of bone metastatic lesions.

Enhancing the tumor-fighting capabilities of NKT cells

The earliest CAR-modified cells were immune T cells. CAR T cells have been proven to be effective in treating certain types of leukemia and lymphoma. However, a number of challenges have been encountered in attempts to treat solid tumors with CAR T cells. Preclinical studies have demonstrated that NKT cells offer a novel approach that may enhance CAR-directed cancer immunotherapy.

"In addition to being able to effectively combat tumors in mouse models, the presence of NKT cells within solid tumors is associated with favorable outcomes in cancer patients," said co-corresponding author Dr. Leonid Metelitsa, professor of pediatric oncology at Baylor and Texas Children’s Hospital and member of Baylor’s Dan L Duncan Comprehensive Cancer Center.

Previous work has shown that NKT cells have a spectrum of anti-tumor activities. For instance, these cells migrate to tumor sites where they kill tumor-associated macrophages, a type of immune cell that can promote tumor growth and metastasis. Moreover, NKT cell activation indirectly promotes an anti-tumor response mediated by two other types of immune cells, NK and T cells.

"We think that NKT cells have substantial potential to serve as valuable contributors to the fight against cancer," said co-corresponding and first author Dr. Andras Heczey, assistant professor of pediatric oncology at Baylor and Texas Children’s and member of the Dan L Duncan Comprehensive Cancer Center. "For the last 10 years, we have been focused on enhancing these cells’ tumor-fighting abilities with the ultimate goal of bringing them to the clinic."

Preparing NKT for clinical trials

The journey to develop NKT cells into a form of immunotherapy involved finding solutions for a number of challenges. For example, NKT cells represent a low percentage of the cells in the blood, so Metelitsa, Heczey and their colleagues developed methods to grow NKT cell populations to clinical scale with high purity.

Although NKT cells can combat tumors in several ways, they all seem to be indirect. "Working with Dr. Gianpietro Dotti at UNC, we gave NKT cells a tool – the CAR – that enables them to attack tumors directly," Metelitsa said. "We also equipped them with IL-15, an additional tool to help them survive in the patient while they fight the tumor."

"The field of CAR cellular immunotherapy for cancer has been focused to date primarily on the manipulation of T lymphocytes," said Dotti, professor and director of cancer cellular immunotherapy at UNC Lindberger Comprehensive Cancer Center. "Based on previous clinical evidence that NKT infiltration within the tumor correlates with favorable clinical outcomes, we decided to leverage this intrinsic property of NKTs and to arm them with an additional bullet – the so-called CAR – to further potentiate their capacity to destroy the tumor."

With all these innovations in hand, the researchers moved on to test CAR NKT cells in patients with neuroblastoma in a clinical trial.

Clinical results

The clinical trial is ongoing, and results from the first three patients with heavily pre-treated, relapsed/refractory metastatic neuroblastoma are presented in this study. The patients were treated with CAR NKT cells, engineered from the patient’s own white blood cells at the Center for Cell and Gene Therapy at Baylor and Texas Children’s. Researchers engineered 95 percent pure NKT cells, a portion of which was armed with CAR-IL15.

"Our initial results show that NKT cells can be expanded to clinical scale with high purity, genetically engineered to express a CAR and IL15, and used to safely treat patients with advanced neuroblastoma," Metelitsa said.

"In addition, we found that CAR-IL15 NKT cells can be detected in the peripheral blood, where they expand postinfusion, traffic to bone metastases and the bone marrow, and exert anti–tumor activity," Heczey said. "We observed an objective response, elimination of at least 50 percent of metastases, in one of the patients."

Dr. Antonio Montalbano and other co-authors from Immunai, a company specializing in single-cell technologies and AI approaches for immunology, applied its state-of-the-art technology platform that allows for the analysis of all genes at the single-cell level in the CAR-NKT patient products. These analyses revealed new information about the heterogeneity of human NKT cells and molecular details of their therapeutic modifications. The researchers discovered nine subsets of NKT cells, and that the CAR receptor seemed to go preferentially to one set identified as cluster 3. Further studies will help understand the implications of these findings.

"Our study shows that it is possible to employ immune cells with natural anti-tumor capabilities and enhance their tumor fighting power with designer synthetic receptors, opening the possibility of applying this strategy to combat hard-to-treat solid tumors," Metelitsa said.

"By leveraging Immunai’s end-to-end platform of artificial intelligence and computational analysis, we were able to zero in on CAR engineered NKT cells from the patients at the single-cell level. The findings from this study are critical toward developing more precise and effective therapies for cancer patients," said Montalbano, genomics technologies lead at Immunai. "We’re looking forward to continuing our research with Baylor with the goal of advancing therapeutic discoveries, accelerating drug development and improving patient outcomes."

The NKT platform developed in this research at Baylor has been licensed to Kuur Therapeutics to advance clinical development.

"The Baylor-Kuur Therapeutics relationship is generating exactly the type of outcomes that we had envisioned at the outset," said Shawn Davis, vice president and chief ventures officer at Baylor. "The modified NKT platform developed in the Metelitsa laboratory is differentiated from other cell therapy platforms, offering novel routes for the treatment of cancers that have posed challenges for immunotherapeutic approaches. The encouraging findings announced today support the potential of NKT platform to provide promising alternatives, particularly for the treatment of solid tumors."

Other contributors to this work include Amy N. Courtney, Simon Robinson, Ka Liu, Mingmei Li, Nisha Ghatwai, Olga Dakhova, Bin Liu, Tali Raveh-Sadka, Cynthia N. Chauvin-Fleurence,Xin Xu, Ho Ngai, Erica J. Di Pierro and Barbara Savoldo. The authors are affiliated with one or more of the following institutions: Baylor College of Medicine, Texas Children’s Hospital, Immunai Inc. and the University of North Carolina at Chapel Hill.

This work was supported by grants/contracts from Alex’s Lemonade Stand Foundation for Childhood Cancer, Kuur Therapeutics, American Cancer Society, Cookies for Kids’ Cancer Foundation, and the Cancer Prevention and Research Institute of Texas (BCM Comprehensive Cancer Center Training Program, RP160283).

On October 12, 2020 Prescient Therapeutics reported that attention for its remarkable progress on the development of a broad pipeline for personalised cancer treatments (Press release, Prescient Therapeutics, OCT 12, 2020, View Source;utm_medium=rss&utm_campaign=shedding-light-on-prescient-therapeutics-ptx-personalised-cancer-treatments [SID1234568605]).

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Personalised cancer treatments of PTX comprises CAR-T and targeted therapies spanning several challenging cancers. Prescient Therapeutics has licensed technologies and collaborations with world-leading cancer centres in the U.S.

Let us deep dive and discuss product pipeline of Prescient Therapeutics-

Universal immune receptor platform, OmniCAR

OmniCAR is a universal immune receptor platform creating next-generation CAR-T therapies. Whilst CAR-T has represented a paradigm-shift in treating cancer, current generation CAR-T is faced with many limitations including safety and flexibility in targeting other cancers.

This is where OmniCAR comes in, creating next-genreation CAR-T that addresses the limitations of current generation CAR-T. OmniCARenables controllable T-cell activity & multi-antigen targeting with a single cell product. The modular CAR system of OmniCAR decouples antigen recognition from the T-cell signalling domain. This platform is based on technology licensed from the University of Pennsylvania and Oxford University.

The universal immune receptor platform allows extraordinary control and flexibility over the current generation CAR-T approaches. With OmniCAR, Prescient will enable its in-house development of next-generation engineered cell therapies. OmniCAR also creates opportunities in collaboration and business development for cell therapy area of Prescient.

Prescient is working with the goal of harnessing innate adaptability and control of OmniCAR for developing novel CAR-T products for new indications. OmniCAR platform improves the cost, time as well as the effectiveness of delivering CAR-T therapies across the world.

READ MORE: Prescient Collaborates With Peter MacCallum Cancer Centre

Moreover, Prescient has several other programs underway to develop new cell therapy methods.

First-in-class drug candidate, PTX-100
PTX-100 disrupts the oncogenic Ras pathway by preventing the activation of Rho, Rac and Ral, leading to cancer cells death. PTX-100 is under clinical development for hematological and solid malignancies under Phase 1b PK/PD basket study.

Currently, PTX is conducting its Phase 1b basket study of PTX-100 at a dose of 2,000 mg/m2.

In this study, Prescient is finding the mutational status of malignancies of each patient and aims to compare this status with any clinical activity. Moreover, the Company is investigating several cancer biomarkers with the objective of identifying patients who may be most likely to respond to PTX-100 therapy.

Novel PH domain inhibitor, PTX-200
A novel PH domain inhibitor PTX-200 inhibits Akt tumor survival pathway. This pathway has a significant role in the development of numerous cancers, including breast & ovarian cancer, along with acute myeloid leukemia (AML). With its novel mechanism of action, PTX-200 specifically inhibits Akt while being comparatively safe. Currently, Prescient is conducting Phase 1b study of PTX-200 & cytarabine in AML patients.

On 3 August 2020, the Company disclosed that it had completed the Phase 1b study for the first cohort at a dose of 25 mg/m2 PTX-200 under the revised study protocol. Currently, Prescient is conducting the Phase 1b study at the increased dose level of 35 mg/m2 PTX-200.

World-renowned leukemia expert Professor Jeffrey Lancet (H. Lee Moffitt Cancer Center, Florida) is leading the AML study.

COVID-19 Antiviral Testing Program
Addition to its personalised cancer treatments Prescient’s two assets are under evaluation against the deadly SARS-CoV-2. On 21 July 2020, Prescient Therapeutics disclosed that its two assets had been nominated as Group 1 priority candidates by Doherty Institute for a COVID-19 antiviral testing program. Both the assets of Prescient are selected on the basis of their potential antiviral properties.

On 12 October 2020, PTX share was trading at A$0.066, with a market capitalisation of A$41.64 million.

To know more about Prescient Therapeutics Limited, click here.

To stay updated with PTX company activities and announcements, please update your details on their investor centre.

Resilient Healthcare Stocks to Beat the Pandemic Heat
With the pandemic continuing to affect the globe, healthcare companies are evaluating their lead compounds for COVID-19 treatment. Future revenue for these stocks depends on the probability of launching an approved treatment in the market.

On October 12, 2020 SQZ Biotechnologies, reported that which already has an alliance with Roche focused on developing new cancer cell therapies, is now looking to sell shares to the public to finance its research of potential new treatments for both cancer and infectious disease (Press release, SQZ Biotech, OCT 12, 2020, View Source [SID1234568458]).

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In paperwork filed with securities regulators late last week, SQZ set a preliminary $75 million goal for its IPO. The Watertown, MA-based biotech has applied for a listing on the New York Stock Exchange under the stock symbol "SQZ."

SQZ (pronounced "squeeze") is developing technology intended to improve cell therapy. Current cell therapies get the therapeutic cargo into a cell via an engineered virus or electroporation, a technique that involves zapping a cell’s membranes open with electricity. The latter approach can damage the cell, impeding its function. SQZ also says in its filing that both techniques are used primarily for delivering nucleic acids to certain cell types and they don’t work well for proteins or small molecules.

On the patient side, cell therapy requires "pre-conditioning," a depletion of immune cells to improve the efficacy of the infused cell therapy. Cell therapies also come with warnings of potentially life-threatening safety risks. Furthermore, both electroporation and viral engineering can lead to downstream biological effects.

SQZ says it can avoid limitations of current cell therapies with a proprietary technology that physically squeezes cells through a microfluidic chip, opening the cell membrane and allowing therapeutic cargo to diffuse inside. The biotech says its approach offers a manufacturing advantage—less than 24 hours production time compared to a month or more for current cell therapies. Cell therapies made with SQZ’s technology don’t require patient pre-conditioning, which shortens hospital stays, and the company says its technology can potentially apply to many more diseases.

The SQZ technology was invented by company founder and CEO Armon Sharei based on the research of his team in the laboratories of Klavs Jensen and Robert Langer at MIT. The company has developed three platforms: one targeting antigen-presenting cells (APCs), another for activating antigen carriers (AACs), and a third for tolerating antigen carriers (TACs). SQZ’s initial focus areas are oncology, infectious disease, and immune disorders. SQZ was an Xconomy Awards 2020 finalist in the "Big Idea" category. Sharei explains his company’s technology in this video (SQZ’s portion starts at 18:18).

Roche began its partnership with SQZ in 2015, the same year that the company licensed its technology from MIT, according to the IPO filing. That alliance aimed to use the SQZ technology to develop cancer cell therapies based on B cells, a type of immune cell. In 2018, the partners amended the pact to include APCs. Under the collaboration agreement, the partners jointly select antigen targets. SQZ is responsible for preclinical research and Phase 1 development of cell therapy candidates. After that, Roche has the option to exercise an exclusive global license to APC products from the collaboration on a product-by-product basis. Those rights only cover cancer. For other indications, or for cancer applications that Roche opts not to pursue, SQZ retains all rights.

The most advanced drug candidate in the alliance, SQZ-PBMC-HPV, comes from SQZ’s APC platform. In preclinical research, SQZ says it was able to squeeze antigens targeting the KRAS cancer protein into two sets of human donor cells. In both, the company observed antigen-specific activation of immune cells, according to the IPO filing. Based on the results, SQZ says it believes cell therapies from its APC platform has potential applications in HPV positive tumors and cancers caused by KRAS mutations, among other oncology applications.

The SQZ drug is currently in Phase 1 testing in in patients with locally advanced and metastatic HPV positive tumors. SQZ expects initial monotherapy data from the study will be reported in the first half of next year. Initial Phase 1 data from tests of that drug as part of a combination therapy are expected in the second half of 2021.

Since its formation, SQZ says it has raised more than $166.6 million from equity investments, most recently a $65 million Series D round in May. The company also says that it has received $94 million in upfront and milestone payments from Roche. Polaris Partners is SQZ’s largest shareholder with just over 14 percent of the company, according to the IPO filing. The second largest shareholder with 9.6 percent is Elbrus Investments, followed by AIG DECO Fund with 7.7 percent.

SQZ says it will apply the IPO proceeds toward the completion of Phase 1 development of its lead therapeutic candidate. Some of the IPO cash will support continued development of SQZ-AAC-HPV, compound from its AAC platform that is another potential treatment for HPV positive tumors. The company says it will also use some of the IPO proceeds for the preclinical research needed to support an application to begin human testing of its treatment candidates for infectious diseases.

On October 12, 2020 Rhizen Pharmaceuticals S. A. (Rhizen), a Switzerland-based privately held clinical-stage biopharmaceutical company, and Curon Biopharmaceutical Limited (Curon), a clinical-stage innovative biopharmaceutical company with facility in Shanghai reported that they have entered into an exclusive licensing agreement for the development and commercialization of Tenalisib, a dual PI3K delta & gamma inhibitor in the Greater China region (Press release, Rhizen Pharmaceuticals, OCT 12, 2020, View Source [SID1234568376]).

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Under the terms of the agreement, Rhizen will receive an undisclosed upfront cash payment and is eligible to receive additional development and commercial milestone payments with an overall deal value of USD 149.5 million plus double-digit royalties on annual net sales of Tenalisib.

Curon obtains the exclusive development and commercialization rights of Tenalisib for Greater China across all oncology indications, and will lead the clinical development in that territory by leveraging its unique expertise in translational research, clinical development and regulatory registration and its extensive research collaboration experience, to accelerate the development of and regulatory approval of this product in Greater China.

Swaroop Vakkalanka, Ph.D., President and Chief Executive Officer of Rhizen Pharmaceuticals stated, "Emerging human clinical data demonstrates that Tenalisib is a differentiated, next-generation, orally active, dual PI3K delta and gamma inhibitor with an excellent safety profile and promising single-agent activity in haeme malignancies. We believe, Tenalisib’s outstanding safety could allow rational combinations with other approved/investigational agents and enable us to unlock the true potential of this class of drugs. Our partnering with Curon is a first step towards achieving this objective and we look forward to the day this novel drug reaches cancer patients in need of new and safe therapies."

"Tenalisib has demonstrated great efficacy in lymphoma patients with outstanding safety profile, in-licensing this product to China would bring more effective and additional treatment options to Chinese cancer patients and greatly benefit these patients. Meanwhile, this will further enrich our diversified pipeline. We are very happy and look forward to closely collaborating with Rhizen to efficiently develop this molecule into an effective medicine to benefit patients not only in China but also around the world as soon as possible." said Zhihong Chen, Ph.D., President of Curon.

Kun Tao from Yafo Capital acted as financial advisor on this transaction for Rhizen.

About Tenalisib (RP6530):

Tenalisib (RP6530) is a highly selective, next-generation, orally active, dual PI3K delta and gamma inhibitor, that is currently in Phase 2 clinical development for hematological malignancies. Tenalisib has been granted US FDA Fast Track & Orphan-Drug Designations for treatment of relapsed/refractory peripheral T-cell lymphoma and cutaneous T-cell lymphoma (R/R PTCL and R/R CTCL).