BDgene completes a round of financing to help gene therapy and achieve a leap from "1 to 100"

On April 28, 2021 Shanghai BDgene Technology Co., Ltd. ("BDgene"), a leading global gene therapy development company with original delivery technology, reported the company has successfully completed a round A financing of 60 million yuan (Press release, BDgene Therapeutics, APR 28, 2021, View Source [SID1234641044]). This financing was led by Tsinghua Holdings Capital , Ennovation Ventures, Longmen Capital, ETP Ventures, Innovation Angel Fund and other institutions followed in the investment. Probe Capital served as the exclusive financial advisor. The funds will be used for preclinical and clinical development of multiple products, laboratory and team building etc. BDgene had previously received a strategic investment from Serve Accurate Faithful Evaluation in June 2019 , and a pre-A round of investment from Keytone Ventures in March 2020.

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According to the data, BDgene was established in 2018 and is an innovative company focusing on the development of in vivo gene editing and in vitro transgene therapy therapies. Since its establishment, the company has attracted much attention from investment institutions and has received investment from many institutions. Why is it sought after by capital? What is the core technology? What are the competitive advantages of leading products?

Gene therapy has become the third revolutionary therapy, but delivery technology is still the most difficult point

For difficult to treat genetic, infectious, and degenerative diseases, gene therapy has the characteristic of "can be cured once". In recent years, it has become the third revolutionary therapy after chemical drugs and antibody drugs.

From the first appearance of the third-generation gene editing technology CRISPR in 2012, to the first in-vivo clinical trials of CRISPR in March 2020, to October 2020 after CRISPR won the Nobel Prize in Chemistry and other major events, the gene editing technology gradually matured , But delivery technology has always been the biggest obstacle in the field of gene editing therapy.

In January 2020, Nobel Prize winner Jennifer Doudna wrote an article in "Nature" magazine that "Delivery remains perhaps the biggest bottleneck to somatic-cell genome editing".

In the past, gene therapy delivery technologies that have been clinically verified by humans in the world mainly include three platforms: Adeno-associated virus, lentivirus vector, and lipid nanoparticles. These platforms have certain drawbacks for gene editing delivery. The ideal gene editing delivery tool needs to have both instantaneous and high-efficiency characteristics to ensure the safety and effectiveness of the treatment.

Create China’s original delivery technology to help gene therapy achieve a leap from "1 to 100"

Dr. Cai Yujia, the founder of BDgene, studied under the professors of Karolinska Institutet in Sweden, the Secretary-General of the Nobel Committee, Professor Thomas Perlmann, and Professor Jacob G. Mikkelsen, Aarhus University, Denmark, and he received a doctorate degree in gene therapy. He has mastered the core technology of gene therapy vectors and has rich experience in the production design and transformation of gene therapy virus vectors. After returning to China, he served as a researcher at the Institute of Systems Biomedicine, Shanghai Jiaotong University. In recent years, BDgene has successfully developed VLP-mRNA, a gene therapy delivery platform that has been clinically validated by humans, to deliver CRISPR/Cas9 mRNA to achieve safe and controllable gene editing therapy in vivo.

BDgene successfully completed the world’s second clinical study of in vivo CRISPR gene editing therapy in early November 2020 (in March 2020, Editas’ Zhang Feng team completed the world’s first case), which is also the world’s first CRISPR antiviral therapy Clinical research. This delivery technology delivers Cas9 mRNA through the lentiviral shell, which combines the high efficiency of viral vectors in infecting cells (in vitro infection rate of 99.8%, in vivo infection rate> 50%) and transient mRNA expression (degraded within 72 hours). Therapeutic effects have been achieved on animal models of different diseases, with technical advantages such as transient expression in vivo, no long-term safety risks associated with AAV expression of CRISPR, no obvious off-target, and low immunogenicity. Currently, with the permission of the Ethics Committee of the Eye, Ear, Nose and Throat Hospital Affiliated to Fudan University, the team of Professor Hong Jiaxu is presiding over the initial safe and controllable clinical application research based on this technology. In the future, this technology will be extended to the treatment of other viral infections, eye diseases and neurological diseases.

Mr. Zhang Yang, Chairman of Tsinghua Holdings Capital said, "The gene therapy industry has the characteristics of huge market space and extremely high technical threshold. In the past, gene editing technology has gradually broken through, but delivery technology is still the biggest bottleneck. In the past 10 years, Dr. Cai Yujia has been devoted to the development of gene therapy Delivery technology, It has successively successfully developed multiple original delivery technology platforms such as virion delivery CRISPR/Cas9 mRNA technology and next-generation lentivirus delivery technology. BDgene’s original viral keratitis treatment product has perfect preclinical animal experiment data. It is currently conducting non-registered human clinical trials in top domestic eye hospitals. BDgene also deployed gene editing technology to treat hereditary, infectious and degenerative diseases, viral vectors to treat blood diseases, and mRNA vaccines. "

Speaking of this Series A financing being exclusively led by Tsinghua Holdings Capital, Mr. Zhang Yang said, "Thank you very much for the trust of BDgene founder Professor Cai Yujia and old shareholders. In the future, he will support the development of the company in the long-term and jointly promote BDgene’s original delivery technology and The product helps gene therapy to achieve a leap from "1 to 100", so that more patients who are refractory to and incurable can really benefit in the future."

Dosing of first patients with RXC004 and Anti-PD1

On April 28, 2021 Redx Pharma (AIM: REDX), the drug discovery and development company focused on cancer and fibrosis, reported that it has successfully initiated dosing of the first patient cohort with a combination of RXC004, the Company’s lead drug candidate, and nivolumab (OPDIVO-Bristol Myers Squibb, an anti-PD-1 antibody) (Press release, Redx Pharma, APR 28, 2021, View Source [SID1234578603]). RXC004 is also currently being evaluated as monotherapy in a Phase 1 clinical study, from which top line results are expected by mid 2021.

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The primary objective of the Phase 1 combination study is to evaluate the safety and tolerability of RXC004 in combination with nivolumab in patients with advanced malignancies (ClinicalTrials.gov Identifier:NCT03447470). The announcement today confirms that the first three patients have initiated treatment with 1mg RXC004 along with nivolumab. The results from the combination study are expected to read out in H2 2021 and will be used to define a dose of RXC004 to be used in combination with standard dose nivolumab in a Phase 2 study in patients with genetically selected microsatellite stable (MSS) metastatic colorectal cancer (MSS mCRC), which is planned to start to recruit patients in H2 2021.

Lisa Anson, Chief Executive Officer of Redx Pharma said: "We are delighted to have initiated dosing of our first patient cohort in our Phase 1 RXC004 and anti-PD1 combination study. We believe that RXC004 has the potential to offer clinical benefit both as a monotherapy and in combination with immunotherapies for patients with Wnt-driven advanced solid tumours.

This important milestone opens our combination study programme, which is an exciting addition to our ongoing Phase 1 monotherapy study which is on track to report headline results by mid 2021"

What is RXC004?
RXC004 is a potent, selective, oral small molecule inhibitor of the enzyme, porcupine, a key activator of Wnt ligands in the Wnt signalling pathway. Aberrant Wnt signalling contributes directly to tumour growth and plays an important role in immune evasion, which leads to resistance to immune checkpoint inhibitors such as nivolumab. By selecting patients with tumours that have high Wnt ligand dependency, such as tumours with mutations in the RNF43 gene and fusions in the RSPO gene family, RXC004 has an opportunity to both directly inhibit the tumour growth and have an immune-enhancing effect to attack the tumour.

Why are we testing anti-PD1 and RXC004 in combination?
Immune checkpoint inhibitors (ICIs) such as anti-PD-1 antibodies have revolutionised the treatment of cancer, but do not work in all patients. Wnt pathway activation can enhance the ability of the tumour to evade destruction by the immune system and could contribute to lack of response to ICIs in these tumours. Our scientists have demonstrated preclinically that RXC004 can block activation of the Wnt pathway and restore the ability of the immune system to fight the tumour. Thus, RXC004 offers potential as a monotherapy or combination therapy, which we are now testing in clinical trials.

RXC004 Clinical trials
RXC004 is currently being investigated in a Phase 1 study, with top line safety and tolerability data as a monotherapy expected by mid 2021, with more detailed data expected to be presented at a conference in H2 2021. Following completion of the monotherapy Phase 1, clinical proof-of concept monotherapy studies in genetically-selected patients with metastatic colorectal cancer, genetically selected pancreatic cancer and all comers biliary cancer are expected to initiate. These studies are in addition to the combination study programme

Seven and Eight Biopharmaceuticals Announces Acceptance of BDB001 in Combination with Pembrolizumab Interim Data to be Presented in a Poster Discussion at the 2021 ASCO Annual Meeting. 

On April 28, 2021 Seven and Eight Biopharmaceuticals Inc., a clinical stage biotechnology company focused on developing proprietary novel immuno-oncology therapies to activate the immune system against cancer, reported the acceptance of an abstract for a poster discussion presentation at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Press release, Seven and Eight Biopharmaceuticals, APR 28, 2021, View Source [SID1234578631]). The abstract details additional interim safety and efficacy results for a phase 1 study of BDB001, an intravenously administered toll-like receptor 7 and 8 (TLR7/8) agonist, in combination with pembrolizumab in advanced solid tumors (NCT03486301).

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"We are excited to share additional information about BDB001 in combination with pembrolizumab at the 2021 ASCO (Free ASCO Whitepaper) Annual Meeting," said Dr. Robert H.I. Andtbacka, Chief Medical Officer, Seven and Eight Biopharma. "This builds upon the promising monotherapy data we reported on recently and helps illustrate the potential for BDB001 in combination with anti-PD-(L)1 therapies in difficult to treat tumors."

The ASCO (Free ASCO Whitepaper) Annual Meeting will be held virtually on June 4-8, 2021 and showcases innovative scientific research in clinical oncology and advancements in cancer therapy.

Details on Seven and Eight Biopharma’s poster discussion presentation:

Abstract Title: BDB001, an intravenously administered toll-like receptor 7 and 8 (TLR7/8) agonist, in combination with pembrolizumab in advanced solid tumors: Phase 1 safety and efficacy results.

Session Title: Poster Discussion Session, Developmental Therapeutics—Immunotherapy

On-Demand Session Release Date and Time: 6/4/2021, 9:00 AM-10:00 AM

Abstract Number for Publication: 2512

This year, ASCO (Free ASCO Whitepaper) reviewed more than 5,400 abstracts for the 2021 ASCO (Free ASCO Whitepaper) Annual Meeting. More information about the meeting may be accessed at View Source According to ASCO (Free ASCO Whitepaper)’s Embargo Policy, abstracts will be released to the public on ASCO (Free ASCO Whitepaper)’s Meeting Library at 5:00 p.m. ET on May 19, 2021.

Results from Calithera Biosciences’ CANTATA Study to Be Presented at 2021 ASCO Annual Meeting

On April 28, 2021 Calithera Biosciences, Inc. (Nasdaq: CALA), a clinical-stage biotechnology company focused on discovering and developing novel small-molecule drugs for the treatment of cancer and other life-threatening diseases, reported that final results from the CANTATA clinical study will be shared in an oral presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2021 Annual Meeting taking place virtually June 4-8, 2021 (Press release, Calithera Biosciences, APR 28, 2021, View Source [SID1234578648]).

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Earlier this year, Calithera announced topline results from the Phase 2 CANTATA trial, which evaluated the efficacy and safety of the company’s glutaminase inhibitor, telaglenastat, in combination with cabozantinib versus placebo with cabozantinib in patients with advanced or metastatic renal cell carcinoma (RCC) who had been treated with one or two prior lines of systemic therapy, including immune checkpoint inhibitor or anti-angiogenic therapy. Results showed that the addition of telaglenastat to cabozantinib did not improve progression-free survival (PFS) in the study population. Additional analyses will be presented.

Following are details of the ASCO (Free ASCO Whitepaper) 2021 presentation of final CANTATA results:

Title: CANTATA: Primary analysis of a global, randomized, placebo (Pbo)-controlled, double-blind trial of telaglenastat (CB-839) + cabozantinib vs. Pbo + cabozantinib in patients (pts) with advanced/metastatic renal cell carcinoma (mRCC) that progressed on immune checkpoint inhibitor (ICI) or anti-angiogenic therapies
Abstract: 4501
Session Title: Genitourinary Cancer—Kidney and Bladder
Session Time: 6/7/2021 8:00am – 11:00am EDT
Presenter: Nizar M. Tannir, M.D., FACP, Professor, Ransom Horne, Jr. Professor for Cancer Research, Department of Genitourinary Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX

Ultragenyx to Host Conference Call for First Quarter 2021 Financial Results and Corporate Update

On April 28, 2021 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel therapies for serious rare and ultra-rare genetic diseases, reported that it will host a conference call on Tuesday, May 4, 2021 at 5pm ET to discuss its financial results and corporate update for the first quarter ended March 31, 2021 (Press release, Ultragenyx Pharmaceutical, APR 28, 2021, https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-host-conference-call-first-quarter-2021-financial [SID1234578668]).

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The live and replayed webcast of the call will be available through the company’s website at View Source To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (International) and enter the passcode 6883837. The replay of the call will be available for one year.