On April 21, 2021 PerkinElmer, Inc., a global leader committed to innovating for a healthier world, reported that it is serving as a provider and co-developer of assays, instrument solutions and expertise for the PROXIDRUGS Consortium which is zeroing in on research for proximity drugs or PROTACs (PROteolysis Targeting Chimeras) (Press release, PerkinElmer, APR 21, 2021, View Source [SID1234578345]). PROTACs are a new class of drugs that take advantage of the body’s own cell protein recycling system to fight disease by tapping into the 80% of disease-relevant proteins that are currently untargeted by today’s available therapeutics. Led by Goethe University in Frankfurt, Germany, the Consortium includes researchers from the Technical University of Darmstadt, the Fraunhofer Institute for Translational Medicine and Pharmacology, and global pharmaceutical companies based in Frankfurt’s Rhine-Main region.

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The research project, starting October 2021, aims to streamline novel drug target validation by putting disease-relevant proteins in close proximity with key enzymes (such as E3 ligases) so that they can be marked for destruction and recycled by the cells’ natural shredding capabilities.

To help in these efforts, PerkinElmer will provide the consortium with no-wash AlphaLISA and HTRF immunoassay technologies and expertise in protein labeling as well as assay design; EnVision multimode plate readers; high content imaging technologies such as the Opera Phenix and Operetta CLS platforms; and data analysis and informatics tools like TIBCO Spotfire software. These PerkinElmer offerings will give PROXIDRUGS scientists a highly sensitive and accelerated discovery platform with rich data delivery and high throughput for both live and dead cell research.

Commenting on this groundbreaking research and collaboration, Alan Fletcher, SVP of Life Sciences at PerkinElmer said, "With only 20% of disease-relevant proteins currently being targeted by classical, small molecule drugs, new research approaches, such as PROTAC drug discovery, hold immense and exciting potential. We are delighted to be able to contribute our expertise and technologies to assist in the innovative work of the PROXIDRUGS Consortium as it seeks new ways to help unlock and tackle the vast majority of proteins behind widespread diseases such as cancer, neurological disorders, and cardiovascular, inflammatory and infectious conditions."

The PROXIDRUGS consortium is led by Ivan Đikić of Goethe University Frankfurt who also commented on the importance of forming alliances across organizations to help advance novel drug discovery and development, "Successful translation of biomedical research requires strong collaboration and convergence of industry and academic expertise. Our work with PerkinElmer within the PROXIDRUGS consortium is a good example of that. PerkinElmer’s expertise and innovation in assay development is of utmost importance and will help to create new insights for our discovery efforts."

The PROXIDRUGS Consortium was recently selected for funding by the German Federal Ministry of Education and Research as part of a highly competitive "Clusters4Future" competition.

On April 21, 2021 Synthesia, the AI video company, that has built and operates the world’s first and largest AI video platform, reported a $12.5M Series A financing round led by Matt Turck from FirstMark Capital with participation from all existing investors (Press release, UCLB, APR 21, 2021, View Source [SID1234578291]). The round is the largest investment to date in the synthetic AI video space.

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Founded in 2017 by technology entrepreneurs Victor Riparbelli, Steffen Tjerrild and world-renowned professors of computer vision Matthias Niessner and Lourdes Agapito, Synthesia is focused on reducing the friction of video creation and making it possible for anyone to create professional-looking videos in minutes, directly from their browser.

The funding will be used to fuel both Synthesia’s rapid enterprise user growth and product development of their AI video generation platform.

On April 21, 2021 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, reported that Manuel Litchman, M.D., President and Chief Executive Officer, and Knut Niss, Ph.D., Chief Technology Officer, will participate in Chardan’s 5th Annual Genetic Medicines Manufacturing Summit, taking place virtually on April 26 and 27, 2021 (Press release, Mustang Bio, APR 21, 2021, View Source [SID1234578310]). The company will participate in a fireside chat on Monday, April 26, 2021, at 3:15 p.m. ET and will also participate in one-on-one meetings during the conference. A webcast of the company’s fireside chat will be available on the Events page of the Investor Relations section of Mustang’s website, www.mustangbio.com, for approximately 30 days after the meeting.

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On April 21, 2021 Centessa Pharmaceuticals reported that it is aiming for the public markets (Press release, Centessa Pharmaceuticals, APR 21, 2021, View Source [SID1234578396]). The company filed on Wednesday to raise up to $100 million in its Nasdaq IPO, but, if recent deals are any indication, it will likely rake in considerably more.

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The news comes a month after chief scientific officer, Moncef Slaoui, M.D., departed the company amidst allegations of sexual assault at his former employer, GlaxoSmithKline.

The proceeds will fund several clinical trials that are already underway, including a phase 3 safety study for lixivaptan, a treatment for autosomal dominant polycystic kidney disease, and a phase 2a study for SerpinC, a treatment for the blood disorders hemophilia A and B, the company said in a securities filing.

RELATED: GSK cuts ties with Slaoui over sexual harassment allegations as CEO Walmsley vows to rename R&D site

The funds will also bankroll future clinical trials, including a pivotal phase 3 trial for lixivaptan; a phase 1 study for ZF887, a treatment for the genetic disorder alpha-1-antitrypsin deficiency, which can cause liver and lung disease; and a phase 2 study for imgatuzumab, an anti-EGFR antibody for the treatment of cutaneous squamous cell carcinoma, the second most common form of skin cancer.

Centessa will also invest in its discovery, manufacturing and R&D capabilities, as well as the design and execution of preclinical and clinical studies for its earlier-stage pipeline. And the capital could go toward acquiring and developing yet more programs, according to the securities filing.

The company came out of the shadows in February, having acquired 11 biotech companies and raised $250 million in series A funding. Its goal was to create an asset-centric drug developer without the R&D inefficiencies that strike "classical pharmaceutical companies," in the words of co-founder Francesco De Rubertis, Ph.D., a partner at Medicxi and chairman of Centessa’s board of directors.

The companies that merged to become Centessa continue to develop their assets under the leadership of the Centessa team and with access to centralized resources such as manufacturing, regulatory and operational support as well as capital.

RELATED: Slaoui’s Centessa adds ex-FDA commish Califf, biotech bigwigs to its board

"This approach encourages an environment where scientific teams are incentivized to maintain an unwavering focus on advancing medicines to key go/no-go inflection points based on data-driven decisions," said Centessa CEO Saurabh Saha, M.D., Ph.D., previously senior vice president of R&D and global head of translational medicine at BMS.

Centessa started out with Saha at the helm and Slaoui, the former co-leader of the U.S. government’s Operation Warp Speed effort and longtime GlaxoSmithKline executive, as chief scientific officer. But Slaoui’s tenure at the new biotech was short-lived. Barely a month after Centessa’s launch, his former employer cut ties with him following allegations from an employee of sexual harassment that occurred several years ago at GSK. A day later, he stepped down from his role at Centessa.

On April 21, 2021 Allogene Therapeutics, Inc., a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) therapies for cancer, reported that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to ALLO-715 in relapsed/refractory multiple myeloma (Press release, Allogene, APR 21, 2021, View Source [SID1234631116]). The FDA granted RMAT designation based on the potential of ALLO-715 to address the growing unmet need for patients who have failed other multiple myeloma therapies.

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"RMAT designation was granted based on our encouraging initial clinical experience in heavily pretreated patients. ALLO-715 demonstrated for the first time that an allogeneic CAR T therapy directed at BCMA can achieve deep clinical responses while eliminating the need for bridging therapy and delays associated with autologous CAR T manufacturing," said Rafael Amado, M.D., Executive Vice President of Research and Development and Chief Medical Officer. "We look forward to completing the UNIVERSAL study and working closely with the FDA as we seek to rapidly advance this important therapeutic alternative to patients with advanced multiple myeloma."

Initial results from the UNIVERSAL study were presented at an oral session of the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December 2020. As part of the Company’s anti-BCMA strategy, the Phase 1 UNIVERSAL study continues to optimize the dosing regimen of ALLO-715 and ALLO-647. The UNIVERSAL study is also enrolling patients to receive ALLO-715 in combination with SpringWorks Therapeutics’ investigational gamma secretase inhibitor, nirogacestat.

Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the development and review processes for promising pipeline products, including cell therapies, that includes all the benefits of Fast Track and Breakthrough designation. An investigational cell therapy is eligible for RMAT designation if it is intended to treat, modify, reverse, or cure a serious or life-threatening disease; and preliminary clinical evidence indicates that the therapy has the potential to address unmet medical needs for that disease. Advantages of the RMAT designation include early interactions with FDA that may be used to discuss potential surrogate or intermediate endpoints and potential ways to satisfy post approval requirements.