Using stem cells to combat osteoarthritis

medicalnewstoday | April 23, 2019

In a recent preliminary study, scientists used stem cells to ease osteoarthritis. Although the trial was small-scale, the results are promising and will pave the way for larger studies. Osteoarthritis of the knee (KOA) occurs when the cartilage — the joint's natural cushioning system — breaks down in the knee. Without this buffer, bones can come into contact with each other, causing pain, stiffness, and a loss of flexibility. According to the Centers for Disease Control and Prevention (CDC), osteoarthritis affects an estimated 30 million people in the United States. Osteoarthritis is predominantly a disease of older age, affecting more than one in 10 people over the age of 60 years. As the population of the U.S. is slowly aging, the number of people with osteoarthritis is likely to increase steadily. Although physical interventions and medications can ease symptoms, there is currently no cure because it is not possible to regrow cartilage. Once KOA has progressed to the end stages, the only option is surgical replacement of the joint.

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With Fabry disease, we're (genetic counselors) perfectly suited to be the one who helps people understand how Fabry disease runs in families and the impact Fabry disease has on family members and understand the impact of who might be at risk that have Fabry disease symptoms. Since Fabray disease is such a sneaky condition and the symptoms are not things you can measure easily, like burning pain in the hands and feet that can't be measured on a nerve conduction test or gastrointestinal issues with diarrhea and constipation.


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PHARMA TECH

Therorna completes Series A financing to accelerate pipeline development and clinical transformation of circRNA-based platform

Therorna Inc. | June 21, 2022

Therorna Inc., a China-based biotech company specialized in the development of cutting-edge circular RNA technology-based new vaccines and therapies, announced the completion of a US$42 million Series A financing round. The round was co-led by a well-known industrial investment firm and MSA Capital, with participation from Sherpa Healthcare Partners, 3H Health Investment, and existing investors Quan Capital and Cenova Capital, demonstrating their continuous commitment to supporting Therorna's growth. With the new funding, Therorna plans to accelerate the build-out of its technology platform and the development of its product pipeline, as well as the clinical trials, regulatory applications, and international collaborations. Therorna was founded in Beijing in April 2021. The company focuses on leveraging its proprietary circRNA technology platform to develop next-generation vaccines and innovative therapies. The company has established a strong pipeline of novel prophylactic and therapeutic drugs aiming to address high unmet medical needs. When combined with the previously completed angel and pre-A rounds, the company has to date raised over US$60 million. "We really appreciate the support of our investors. As the world continues to suffer from the COVID-19, scientists are obliged to shoulder the responsibility to provide scientific solutions to end this pandemic. We are devoted to accelerating the clinical application of our circRNA-based vaccines against SARS-CoV-2 and its variants." The scientific founder of Therorna, Professor Wensheng Wei Therorna Chief Executive Officer, Dr. Daniel Tang commented, "I am very grateful for the support of our new and existing investors in this financing round. The successful completion of this round will play a key role in helping Therorna accelerate the development of a new broad-spectrum COVID-19 vaccine and the discovery of additional novel therapies for a variety of diseases. Our team will continue to spare no effort to advance the circRNA platform technology to develop the next-generation of breakthrough therapies and quickly deliver meaningful social benefits." MSA Capital founder and managing partner Yu Zeng added, "We are confident in the potential of circRNA technology for drug development in the area of protein expression and regulation. Compared with linear RNA, circRNA boasts breakthroughs in expression, stability, immunogenicity, and production. In terms of the technology, Chinese scientists have proven their internationally recognized capabilities at innovation and efficient transformation. MSA Capital is impressed by Therorna's strong team of scientists and entrepreneurs. As a serial entrepreneur, Professor Wei has deep expertise in gene editing and circRNA technology application, and Therorna's management team has rich experience in drug R&D and industrial management. MSA Capital will make use of its resources to support the expansion of Therorna in international markets." Sherpa Healthcare Partners investment VP Dr. Changzu Cai said, "mRNA drugs have achieved notable success in the application of COVID-19 vaccines, but challenges in terms of side effects remain to be addressed. Linear RNA is easily degraded and its application beyond vaccines is still under exploration. Thanks to its unique structure, the circRNA as a tool for protein expression has been proven to maintain a relatively stable protein expression level in vivo for a longer time, which is expected to further expand the application scenarios of RNA therapy. The team led by Professor Wei is leveraging their experience in applying circRNA as a gene editing tool to support the development of the circRNA expression tool. The proprietary circularization method developed by Therorna has demonstrated its advantages in terms of dosage, and activation of cellular immunity by being first applied in COVID-19 vaccines. We are pleased to continue supporting the efforts of Wei Lab in converting innovations into benefits for patients and communities, while looking forward to transforming Therorna into a platform-based circRNA technology-focused company with an international presence." 3H Health partner Dr. Minchuan Wang said, "circRNA has better stability and long-lasting expression with a potential for creating a new RNA therapy platform. The combination of Professor Wei's team with strong technical expertise in circRNA and the management team established at an earlier stage of the company with members having rich experience in promoting the adoption of new technologies will help accelerate the clinical transformation of circRNA. We are honored to support Therorna in this financing round, and we will work with the company's team, and their new and existing investors, to help the company assume a leadership role in the realm of circRNA, providing patients with pioneering therapies and addressing unmet clinical needs." Exclusive financial advisor for this transaction and CEC Capital Managing Director, Echo Zhang commented, "circRNA has attracted widespread attention as an emerging technology for RNA drugs. Therorna is expected to take the lead in advancing the technology into clinical applications. We are honored to help Therorna complete this round of financing, and we believe that Therorna will lead the development of the global circRNA sector." About MSA Capital Founded in 2014, MSA Capital is an independent global private equity and venture capital firm with over $2.0 billion under management. The firm manages capital from the world's top sovereign wealth funds, international asset managers, pension funds, university endowments, fund of funds and family offices, as well as China's leading new economy entrepreneurs. MSA actively partners with outstanding entrepreneurs in China and other technology markets to build leading, innovative companies that aim to deliver strong financial returns and meaningful, positive impact. The firm is headquartered in Beijing, with resident teams in Shanghai, Hong Kong, Singapore, Middle East, North Africa and the United States. About Sherpa Healthcare Partners Sherpa Healthcare Partners ("Sherpa") was founded in 2018, the founders previously worked together in one of China's leading venture capital firms, built and led its professional medical investment team since 2011, set up the dedicated healthcare investment fund and responsible for healthcare investment. The founders have been teamed together in close co-operation, built on shared values, mutual trust, and complementary capabilities. Sherpa has now become an influential healthcare VC firm in the industry. Sherpa has invested in leading companies in many key areas, such as Pharma, GeneTech, MedTech and Medical services. From 2011 to 2020, the team has gone through 4 vintage year cycles of multiple U.S dollar funds and RMB funds and made 100 investments. Sherpa has the honor to work with many outstanding entrepreneurs and grow together with them. About 3H Health Investment 3H Health Investment is a Hong Kong and Shanghai based healthcare venture capital firm. It focuses on unmet medical needs and invests in emerging fields of science and medicine to deliver breakthroughs to patients. Leveraging its extensive resources with clinical institutions, industry partners and policy institutes, 3H builds leading innovative healthcare companies together with its partners. About Quan Capital Quan capital is a life sciences venture capital firm with strong China expertise and global capabilities. We discover, incubate, and grow next-generation life science companies in early and growth stage, worldwide. Our portfolio companies pioneer differentiated therapies and enabling technologies to address major human diseases with high unmet medical needs. Quan has offices in Shanghai, Menlo Park and Boston. Our investment professionals combine their strong expertise in both science and business with their diverse experiences in global drug development and healthcare investments, and they leverage their broad network worldwide to help maximize the company's value across geographies and development stages. Visit www.quancapital.com to learn more about us. About Cenova Capital Cenova Capital is one of the earliest healthcare funds dedicated to early and growth-stage investments in the life sciences and healthcare sectors in China. Since inception in 2010, Cenova Capital now has six funds under management with investments in over 80 companies in pharmaceuticals, medical devices,healthcare services and digital heath. Cenova Capital's investors are mainly large domestic and international institutional investors including Asian sovereign wealth investors, government institutions, insurance companies, financial institutions and multinational companies. About CEC Capital CEC Capital is a leading Chinese FA with a focus on the global TMT, consumption and health sectors. In addition to investment banking, CEC Capital is managing a RMB fund and a dollar fund with offices in Beijing, Shanghai, Los Angeles and San Francisco, and it also has a license to the US securities market. The healthcare industry is an advantageous industry that CEC Capital is concerned about. Meanwhile, CEC Capital has the largest and most professional health investment banking team in China with the most complete industry chain.

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PHARMACY MARKET

CASI PHARMACEUTICALS ANNOUNCES CNCT19 (CD19 CAR-T) GRANTED ORPHAN DRUG DESIGNATION BY THE U.S. FDA

CASI Pharmaceuticals, Inc. | January 19, 2022

CASI Pharmaceuticals, Inc. a U.S. biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products, announced today, that the U.S. Food and Drug Administration as granted Orphan Drug Designation for CNCT19, an investigational CD-19 directed CAR-T therapy, for the treatment of patients with Acute Lymphoblastic Leukemia. CNCT19 is currently being developed independently by Juventas to meet the urgent clinical needs of patients with hematologic malignancies globally. The National Medical Products Administration has granted CTA approval for CNCT19 in two indications in Nov. 2019. Currently, the Phase II clinical trials of CNCT19 are in progress. Positive clinical data for adult and pediatric patients with relapsed/refractory B-ALL has been presented at the December 2021 ASH annual meeting, which further demonstrated its safety and efficacy profile. CNCT 19 is expected to be the first domestic CD19 directed CAR-T product in China with independent intellectual property rights. "Our partner Juventas continues to make encouraging progress in developing their CD19 CAR-T therapy. The Orphan Drug Designation, from the FDA, and the Breakthrough Designation status, granted by the China Center of Drug Evaluation (CDE) in December 2020, represent significant milestones that demonstrate our belief that CNCT19's commercialization will not only be successful in China, but potentially on a global scale. CASI has worldwide co-commercial rights of CNCT19, and will start the global commercialization process according to CNCT19's regulatory progress outside China." Dr. Wei-Wu He, CASI's Chairman, and CEO About Juventas Juventas is a biopharmaceutical company headquartered in China dedicated to the development and commercialization of cell therapies globally. Utilizing innovative and integrated technology platforms, the company has developed a diverse pipeline of cellular immunotherapies for treatment of hematological malignancies, solid tumors, and other non-oncological conditions both in China and globally. At present, the company is conducting two pivotal clinical trials of CNCT19 for treating adult r/r-B-ALL and r/r-B-NHL in China. CNCT19 has the potential to become the first launched domestically developed CD19 CAR-T therapy in China and the first CAR-T product for the treatment of adult R/R B-ALL in China. About CASI Pharmaceuticals CASI Pharmaceuticals, Inc. is a U.S. biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products in China, the United States, and throughout the world. The Company is focused on acquiring, developing and commercializing products that augment its hematology oncology therapeutic focus as well as other areas of unmet medical need. The Company intends to execute its plan to become a leader by launching medicines in the greater China market leveraging the Company's China-based regulatory and commercial competencies and its global drug development expertise. The Company's operations in China are conducted through its wholly-owned subsidiary, CASI Pharmaceuticals (China) Co., Ltd., which is located in Beijing, China. The Company has built a commercial team of more than 100 hematology oncology sales and marketing specialists based in China.

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BUSINESS INSIGHTS

Avidien Technologies Receives $4M Investment from BroadOak Capital Partners

Avidien Technologies | February 02, 2022

Avidien announced a new financing round of $4M from BroadOak Capital Partners. The investment will help Avidien expand its product roadmap, scale commercial production, and better serve research and pharmaceutical clients. "The partnership with BroadOak is exciting and ushers in the next phase for Avidien by creating the opportunity to quickly scale up the business with investments in new products, sales, marketing, and manufacturing." Avidien CEO and Founder Rich Cote Avidien's flagship product, the microPro 300, provides precision semi-automated 96 and 384 channel pipetting to hundreds of biotech, medical testing, and research laboratories worldwide. The compact and portable liquid handling platform has built a loyal customer base thanks to its innovative design, intuitive interface, and industry-leading capabilities. The microPro 300 fills gaps in automation workflows by streamlining repetitive pipetting tasks and ensuring accurate, repeatable liquid transfers. BroadOak Capital Partners is a life sciences focused financial institution with a track record of supporting companies at the forefront of major industry trends. "Aviden's affordable pipette automation platform sets a new standard for common research methods and is at the cutting edge of laboratory automation technology. We're proud to partner with the Avidien team to help them achieve even greater growth," said Bryan Poltilove, Operating Partner at BroadOak Capital Partners. Mr. Poltilove is also joining Avidien's Board of Directors to guide the company's next phase of business expansion. About Avidien Technologies Avidien Technologies is a Massachusetts based company that manufactures innovative and precision liquid handling solutions that are deployed in life science labs around the world. About BroadOak Capital Partners BroadOak Capital Partners, with headquarters in Washington, DC and San Francisco, is a life sciences focused financial institution that provides direct investment and investment banking services to companies in the research tools and consumables, diagnostics, biopharma services, and adjacent markets. BroadOak has led or participated in investments in more than 50 companies across multiple funds and investment vehicles.

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PHARMA TECH

Aquavit Further Prevails Against Counterfeiters in U S Following its Recent Victory in Europe

Aquavit Pharmaceuticals, Inc | March 22, 2022

Aquavit Pharmaceuticals, Inc continues to succeed in its intellectual property cases against counterfeiters both domestically and abroad. Following a recent win against counterfeiter U-BioMed, Inc. across the European Union, the U.S. Southern District Court of New York adopted Judge Lehrburger's recommendations that any sanctions related to U-BioMed's deletion of its Instagram account, which blatantly exploited Aquavit's AQUAGOLD product illegally, be folded into the pre-existing sanctions requiring U-BioMed to pay Aquavit's reasonable legal fees and costs associated with the sanctions-related motions. The Court further reinforced Lehrburger's preview award ordering U-BioMed to pay attorney fees and costs to Aquavit in compensatory damages. U-BioMed's continuous violation of the court's order in 2019 has to date accumulated the court's coercive sanction to millions of dollars. "The depths to which counterfeiters have gone to exploit Aquavit's patented technology, confuse customers, and attempt to trick the legal system(s) is criminal, especially with FDA regulated medical devices. It is of the utmost importance that we protect our physicians from liability and misinformation and continue to keep our patients safe." Sobin Chang, CEO of Aquavit Aquavit's victorious claim against U-BioMed with the European Union Intellectual Property Office (EUIPO) has set a significant milestone. The court acknowledged that U-BioMed knowingly and intentionally filed an infringing trademark application with the EUIPO for the mark "AQUAGOLD Fine Touch". "The counterfeits appear almost identical to the naked eye; however, they do not contain the microchannels as they claim and are often made of regular steel that are not biocompatible under the FDA's medical device material guidelines. Furthemore, they infringe on our 'repeated motion' drug delivery utility patent (U.S. Patent No . 10,980,865) for any drugs, components or biologics." says Dr. David Shafer, Aquavit's Chief Medical Technology Officer. The EUIPO has declared U-BioMed's actions to be willful and done purposefully in an effort to confuse consumers with Aquavit's already existing AQUAGOLD products being sold globally. The EUIPO issued a cancellation of U-BioMed's infringing mark, upheld Aquavit's application for a declaration of invalidity, and held U-Biomed accountable for the legal costs in the European Union. About Aquavit Aquavit is a high-tech pharmaceutical company that provides a comprehensive range of innovative pharmaceutical, biotech and medical device technologies. Aquavit focuses on personalized medicine to improv.

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Spotlight

With Fabry disease, we're (genetic counselors) perfectly suited to be the one who helps people understand how Fabry disease runs in families and the impact Fabry disease has on family members and understand the impact of who might be at risk that have Fabry disease symptoms. Since Fabray disease is such a sneaky condition and the symptoms are not things you can measure easily, like burning pain in the hands and feet that can't be measured on a nerve conduction test or gastrointestinal issues with diarrhea and constipation.

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