Forge Biologics, a gene therapy-focused contract development and manufacturing organization, reported that the closing of a $120 million Series B financing (Press release, Forge Biologics, APR 29, 2021, View Source [SID1234578742]). The financing was led by RA Capital Management with participation from Perceptive Advisors and related affiliates, Surveyor Capital (a Citadel company), Octagon Capital, and Marshall Wace. Existing investors Perceptive Xontogeny Venture Fund and Drive Capital also participated. In connection with the financing, Matthew Hammond, Ph.D., of RA Capital, and Fred Callori of the Perceptive Xontogeny Venture Fund will join the Company’s Board of Directors.

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"We are very pleased to be working with RA Capital and a strong syndicate of top-tier life sciences investors who share our vision as we now advance our growth as a global gene therapy manufacturing and development company," said Timothy J. Miller, Ph.D., CEO, President and Co-Founder of Forge Biologics. "We have built Forge into a focused gene therapy development engine with a firm commitment to helping our clients provide potentially life-saving gene therapies to patients. We believe that focusing entirely on gene therapy will allow us to best serve our clients and patients by efficiently delivering high quality product."

Forge will use the proceeds of this Series B financing to accelerate the expansion of its AAV manufacturing CDMO capabilities with cGMP production capacity, as well as operate its subsidiaries that are advancing novel AAV gene therapy programs. Forge brings a patients-first approach to accelerate the development of transformative medicines for those who need them most and is addressing the growing demand for gene therapy manufacturing capacity. Through its currently-operational 175,000 square foot cGMP facility in Columbus, Ohio, dedicated to AAV viral vector manufacturing, Forge offers end-to-end manufacturing services, including research and toxicology grade AAV production, to accelerate gene therapy programs from preclinical through clinical and commercial stage manufacturing.

"The success of complex biologics like AAV is exhausting existing manufacturing capacity around the industry. This financing will help to address this industry-wide capacity shortage by properly capitalizing an emerging gene therapy-focused CDMO capable of producing high quality cGMP product for its clients," said Matthew Hammond, Ph.D., principal at RA Capital Management. "We are confident that Forge’s experienced team will become the trusted partner of innovative therapeutics companies, working collaboratively with clients to successfully deliver AAV manufacturing solutions."

Through its subsidiaries, Forge is also working to advance a proprietary pipeline of novel gene therapies, including its lead program FBX-101 for the treatment of patients with Krabbe disease, a first-in-human gene therapy utilizing an adeno-associated virus (AAV) to deliver a functioning copy of the GALC gene intravenously to cells in the central nervous system (CNS) and peripheral organs.

Chardan Capital Markets acted as exclusive placement agent for the offering, with Ice Miller acting as legal advisor.

Adcendo, a biotech company developing antibody-drug conjugates (ADCs) for the treatment of cancers, reported that the successful closing of a EUR 51 million (US$ 62 million) Series A financing (Press release, ADCendo, APR 29, 2021, View Source [SID1234578741]). The investment was led by Novo Seeds, the early-stage investment and company creation team of Novo Holdings, and Ysios Capital, along with RA Capital Management, HealthCap and Gilde Healthcare. The company was initially incubated and funded at the BioInnovation Institute (BII).

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The new financing, which is the largest Series A financing for a Danish biotech company, will be used to establish a pipeline of ADCs directed at novel cancer targets and to bring the lead program targeting the novel cancer target uPARAP/Endo180 to proof of concept in patients.

Commenting on the financing, Henrik Stage, Chief Executive Officer of Adcendo, said: "In the last few years the ADC modality has delivered promising approvals of new drugs as well as significant commercial transactions. We are excited that we have secured this major financing from top tier investors and are looking forward to delivering on our vision of bringing new innovative treatments to cancer patients."

John Haurum, Chairman of the Board of Adcendo, added: "With this funding we will be able to bring our first program – the uPARAP program – all the way to proof of concept in patients, translating the basic research done at The Finsen Laboratory benches, incubated through the BII’s Creation House Program, to new, real-life treatments."

uPARAP is a unique novel cancer target overexpressed on the cell surface of several cancers. Being a collagen scavenger receptor that possesses constitutively active and highly efficient internalization and recycling properties, it has been demonstrated to play a role in tumor invasion. The expression and biological mechanisms of uPARAP makes it ideal for an ADC approach as it may be used as a cancer-associated "drug internalization pump" to bring conjugated drugs directly into the cancer cells.

The uPARAP collagen scavenger receptor has been found to be overexpressed by cancer cells in several indications with high unmet needs including soft tissue sarcoma, glioblastoma multiforme, triple-negative breast cancers, leukemia and osteosarcoma, as well as by stromal cells in several high prevalence cancers with substantial stromal tissue content, such as prostate, breast and pancreatic cancer.

In addition to the uPARAP program, Adcendo will build a pipeline of additional novel cancer targets ideally suited to ADC approaches.

Jeroen Bakker, Principal at Novo Seeds, commented: "Novo Seeds is focused on building world class companies that are developing innovative treatments for patients with unmet medical needs. We are proud to have been involved with the company since its early days and are very impressed with the progress achieved to date. We are very pleased to now co-lead this strong investment, building on the founders’ early-stage research at The Finsen Laboratory to develop Adcendo into a world leading ADC player with a pipeline of exciting novel ADC drugs."

Joël Jean-Mairet, Managing Partner at Ysios Capital, added: "Adcendo is taking the ADC approach to the next level by focusing on targets that are professional internalizers and have exquisite tumor selectivity. We are very pleased to support this financing."

The Board of Adcendo includes: John Haurum, Chair; Jeroen Bakker, Novo Seeds; Joël Jean-Mairet, Ysios Capital; Sanne de Jongh, Gilde Healthcare; Reza Halse, RA Capital Management; Mårten Steen, HealthCap and Lars Engelholm of Adcendo.

On April 29, 2021, Acceleron Pharma reported that Bristol Myers Squibb ("BMS") has announced net sales of REBLOZYL (luspatercept-aamt) to be approximately $112 million for the quarter ended March 31, 2021. As previously disclosed, under the collaboration agreement between Acceleron Pharma Inc. (the "Company") and BMS for REBLOZYL, the Company is eligible to receive tiered royalty payments from BMS on net sales of REBLOZYL in the low-to-mid 20% range. The Company expects to report royalty revenue of approximately $22.4 million from net sales of REBLOZYL in the quarter ended March 31, 2021.

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This preliminary unaudited revenue estimate is the responsibility of management and is subject to the completion of the Company’s customary quarter-end financial closing procedures, including management’s review and finalization, as well as review procedures by the Company’s independent registered public accounting firm, which have not yet been completed. During the course of the Company’s review process, items may be identified that would require it to make adjustments, which could result in material changes to the Company’s preliminary unaudited estimated financial results. Consequently, this revenue estimate should not be viewed as a substitute for the Company’s earnings release and the Quarterly Report on Form 10-Q for the quarter ended March 31, 2021.

The information contained in this Item is being furnished and shall not be deemed "filed" for any purpose, and shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Securities Exchange Act of 1934, as amended, regardless of any general incorporation language in any such filing.

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."

On April 28, 2021, Akeso, Inc. reported that, the Center for Drug Evaluation ("CDE") of the National Medical Products Administration (NMPA) of the People’s Republic of China (the "PRC") agreed to start the evaluation of the first-in-class novel drug Cadonilimab (PD-1/CTLA-4 bi-specific antibody, research and development code: AK104), which is an immuno-oncology therapy independently developed by the Company, plus platinum-based chemotherapy in combination with/without bevacizumab for a randomized, double-blind, placebo-controlled phase III clinical trial for first-line treatment of persistent, recurrent or metastatic cervical cancer (Press release, Akeso Biopharma, APR 28, 2021, View Source [SID1234633507]). This is the first phase III clinical trial of dual immunotherapy for first-line treatment of cervical cancer in the PRC.

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Recently, the Company successfully held a seminar of this global phase III clinical trial attended by experts and scholars of world-renowned gynecological oncology research institutions from China, the United States, Europe and Australia. The seminar reviewed the current status of cervical cancer treatment in the world, summarized the results of early stage clinical trials of Cadonilimab on several tumor indications, and discussed in detail the framework proposal of the global phase III clinical trial of Cadonilimab.

President & CEO of Akesobio, Michelle Xia, PhD, said, "Cadonilimab has demonstrated exceptional clinical efficacy in early stage clinical trials on cervical cancer. Based on this, the Company will further carry out the global phase III clinical trial for the first-line treatment of cervical cancer, which shows the excellent clinical operation efficiency of the company and will be beneficial to more patients suffering from cervical cancer around the world. The Company believes that Cadonilimab will become the first PD-1/CTLA-4 bi-specific antibody novel drug approved for market launch in the world."

Because of its satisfactory clinical data, Cadonilimab obtained fast track designation from the Food and Drug Administration of the United States (FDA) for treating patients with recurrent or metastatic cervical cancer after standard therapies in August 2020. Cadonilimab was also approved by the CDE for the inclusion in the "Breakthrough Therapy Designation" list for treating patients with recurrent or metastatic cervical cancer after standard therapies in October 2020. Patient enrollment of the registrational phase II clinical trial of Cadonilimab for the treatment of recurrent or metastatic cervical cancer was completed in January 2021 in the PRC.

INFORMATION ABOUT CADONILIMAB (PD-1/CTLA-4 BI-SPECIFIC ANTIBODY)

Cadonilimab (AK104) is a novel, potential next-generation, first-in-class bi-specific PD-1/ CTLA-4 immuno-oncology backbone drug independently developed by the Company, and its major indications include liver cancer, cervical cancer, lung cancer, gastric cancer, esophageal squamous cell cancer and nasopharyngeal carcinoma. The preliminary research data of cervical cancer, gastric cancer and other tumors shows that, as compared with the combination therapy of PD-1 and CTLA-4, Cadonilimab has much lower toxicity and demonstrated promising safety profile and efficacy.Our AK104 project has been incorporated in the Major New Drug Innovation Program under the 13th Five-year Plan for Major Technology Project (The 13th Five-Year Plan for Major Technology Project) issued by National Health Commission and Ministry of Science and Technology in 2017 and has been enlisted in the 2017 Pearl River Talent Program of Guangdong Province — Introduction of Innovation and Entrepreneurship Team Support Program (2017 Guangdong Province "Pearl River Talent Program" Introduction of Innovation and Entrepreneurship Team Support Project). It was also jointly rated by China Medical Biotechnology Association and Chinese Medicinal Biotechnology as one of the 2017 Top Ten Medicinal Biotechnology Advancements in China (2017 Top Ten Medicinal Biotechnology Advancements in China).It was also jointly rated by China Medical Biotechnology Association and Chinese Medicinal Biotechnology as one of the 2017 Top Ten Medicinal Biotechnology Advancements in China (2017 Top Ten Medicinal Biotechnology Advancements in China).It was also jointly rated by China Medical Biotechnology Association and Chinese Medicinal Biotechnology as one of the 2017 Top Ten Medicinal Biotechnology Advancements in China (2017 Top Ten Medicinal Biotechnology Advancements in China).