CRISPR Gene Editing Ability Improved by Specific Modifications of tracrRNA

GEN | November 11, 2019

Scientists at the City of Hope believe they may have found a way to sharpen the fastest, cheapest, and most accurate gene editing technique, CRISPR-Cas9, so that it can more successfully cut out undesirable genetic information. This improved cutting ability could one day fast-track potential therapies for HIV, sickle cell disease, and, potentially, other immune conditions. “Our CRISPR-Cas9 design may be the difference between trying to cut a ribeye steak with a butter knife versus slicing it with a steak knife,” said Tristan Scott, PhD, lead author of the study and a staff research scientist at City of Hope’s Center for Gene Therapy. “Other scientists have tried to improve CRISPR cutting through chemical modifications, but that’s an expensive process and is like diamond-coating a blade. Instead, we have designed a better pair of scissors you can buy at any convenience store.” The study, “Improved Cas9 activity by specific modifications of the tracrRNA,” published in Scientific Reports is the first time scientists have systematically gone through the guide RNA sequence to change it and improve CRISPR-Cas9 technology, Scott said. The Kevin Morris Lab at City of Hope has filed a patent application claiming this improved CRISPR-Cas9 design, which could result in a doubling of activity but the exact amount was dependent on the target site, Scott said.

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INDUSTRIAL IMPACT

Heska to Acquire LightDeck Diagnostics to Invest in Manufacturing Capabilities and Accelerate Research and Development Efforts

LightDeck Diagnostics and Heska | September 13, 2022

Heska Corporation a leading global provider of advanced veterinary diagnostic and specialty solutions, announced today that the Company has entered a definitive agreement to acquire 100% of the share capital of MBio Diagnostics, Inc. d/b/a LightDeck Diagnostics a pioneer in innovative planar waveguide fluorescence immunoassay diagnostics with strong manufacturing and specialized production capabilities. Founded in 2009 and headquartered in Boulder, Colorado, LightDeck has developed a scalable and innovative platform for rapid and accurate point of care diagnostics. LightDeck's 80-member team includes industry veterans, engineers, and scientists, with deep technical expertise in assay development, manufacturing, and software development, who have collaborated with commercial, academic, and government partners worldwide to build an extensive intellectual property portfolio of 16 US patents and 15 international patents with broad applications, including in veterinary diagnostics, water testing, and human in-vitro diagnostics use. LightDeck's planar waveguide technology optimizes laser activated fluorescence and microfluidics to provide point of care cartridge-based testing without compromise, enabling reference lab-quality results in minutes with inexpensive, quantitative, and easy-to-use functionality. LightDeck's current Boulder location includes fully equipped laboratories for assay development and controlled manufacturing capabilities that support its new, state-of-the-art, highly automated facility in Longmont, Colorado, anticipated to go online in 2023 to dramatically scale-up capacity, production, and research and development. "We are thrilled to welcome the entire LightDeck team to Heska. We have known and closely followed their success for years and we are happy to fully join with them today as one team focused on supercharging their great work. LightDeck brings to Heska best-in-breed, highly scalable manufacturing and world-class assay development to help drive our strategies for winning at innovation and winning at scale," commented Kevin Wilson, Heska's Chief Executive Officer and President. "LightDeck was central in developing Heska's leading immunodiagnostic platform, Element i+®, including its current menu of Total T4, cTSH, Cortisol, and Progesterone. Having proven our capability, we now take this next important step as we aim to drive production of Element i+® analyzers and cartridges at scale and to accelerate key test menu expansion, such as our recently announced Nu.Q® Vet Cancer Screen, cCRP, SDMA, infectious diseases, and more, in single and multi-plex formats that are substantially better than the competition. We expect to drive gross margin improvement with new manufacturing capabilities and scaling across the business. The addition of LightDeck is a great strategic, financial, and cultural fit with Heska as we continue our march to the top of the innovation and manufacturing competition for the most valuable diagnostics," concluded Mr. Wilson. "This is a terrific outcome for LightDeck and Heska and their shareholders, employees and commercial partners. We have known and worked with Heska for years and we greatly admire the Heska organization. Since its founding in 2009, LightDeck has been dedicated to revolutionizing testing at the point of care- for faster, more accurate, less expensive, multi-plex testing of the most important diagnostic tests. We have developed one of the most innovative and technologically-advanced assay platforms in the world, which has led to our recent addition to Fast Company's Annual List of the World's 50 Most Innovative Companies for 2022. I am very proud of the work our team has accomplished over the last 13 years." LightDeck's Co-Founder and Chief Technology Officer, Mike Lochhead, went on to say, "We are excited to now enter this next phase with Heska to dramatically expand our reach and scale. What we do at LightDeck matters a great deal. With Heska, we go confidently into our next commercial, manufacturing, and development chapters, strongly engaged, highly supported, and clearly set-up for long-term success and growth." Nick Traggis, LightDeck's Chief Executive Officer About Heska Heska Corporation manufactures, develops and sells advanced veterinary diagnostics, informatics, and specialty healthcare solutions through its two business segments: North America and International. Both segments include Point of Care Lab testing instruments and consumables, digital imaging products, software and services, data services, allergy testing and immunotherapy, and single-use offerings such as in-clinic diagnostic tests and heartworm preventive products. The North America segment also includes private label vaccine and pharmaceutical production for third-parties, primarily for herd animal health. About LightDeck Diagnostics LightDeck Diagnostics believes in a new approach to healthcare, where quick and accurate diagnostic tests will be run wherever and whenever they are needed. The company's proprietary LightDeck® platform combines an advanced laser waveguide with novel materials and patented manufacturing techniques to deliver lab-quality results anywhere, in minutes. The LightDeck platform is achieving success in veterinary diagnostics and for environmental testing. LightDeck is also developing a portfolio of in vitro diagnostic panels. The company is based in Boulder, Colorado, and is recognized as Colorado BioScience Association's Company of the Year, 2021.

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MEDTECH

Acorda Therapeutics Announces $16.5M Award and Royalty/Supply Relief in AMPYRA® Arbitration Case

Acorda Therapeutics, Inc. | October 17, 2022

Acorda Therapeutics, Inc. announced that a three-judge arbitration panel has issued a final decision in a dispute with Alkermes PLC regarding licensing royalties relating to AMPYRA. Acorda was awarded $15 million plus prejudgment interest of $1.5 million from Alkermes. In addition, as a result of the panel’s ruling, Acorda will no longer have to pay Alkermes any royalties on net sales for license and supply of AMPYRA, and Acorda is now free to use alternative sources for supply of AMPYRA, which the Company has already secured. “This award will allow Acorda to obtain more competitive market rates for the supply of AMPYRA, significantly reducing our cost of goods and meaningfully increasing the product’s value to the company. This will also help Acorda to continue to provide its customary high level of support for Ampyra, ensuring that as many people with MS as possible may benefit from it.” Ron Cohen, M.D., Acorda’s President and CEO Acorda filed an arbitration demand with the American Arbitration Association in July 2020 after the parties were unable to resolve their dispute over license and supply royalties following the 2018 expiration of an Alkermes patent relating to AMPYRA. About Acorda Therapeutics Acorda Therapeutics develops therapies to restore function and improve the lives of people with neurological disorders. INBRIJA is approved for intermittent treatment of OFF episodes in adults with Parkinson’s disease treated with carbidopa/levodopa. INBRIJA is not to be used by patients who take or have taken a nonselective monoamine oxidase inhibitor such as phenelzine or tranylcypromine within the last two weeks. INBRIJA utilizes Acorda’s innovative ARCUS® pulmonary delivery system, a technology platform designed to deliver medication through inhalation. Acorda also markets the branded AMPYRA® Extended Release Tablets, 10 mg.

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CELL AND GENE THERAPY

Rocket Pharmaceuticals to Acquire Renovacor, Extending Leadership in AAV-based Cardiac Gene Therapy

Rocket Pharmaceuticals, Inc. | September 21, 2022

Rocket Pharmaceuticals, Inc. a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, and Renovacor, Inc. a biotechnology company focused on delivering innovative precision therapies to improve the lives of patients and families battling genetically-driven cardiovascular and mechanistically-related diseases, today announced a definitive agreement under which Rocket will acquire Renovacor in an all-stock transaction for an implied value of approximately $2.60 per share, based on the volume weighted average trading price of Rocket shares of $15.51 for the 30 trading days through and including Monday, September 19, 2022. The boards of directors of both companies have unanimously approved the transaction, which is currently expected to close by the first quarter of 2023. “The acquisition of Renovacor aligns with our strategy to expand our leadership position in AAV-based gene therapy for cardiac disease and gives us a perfect opportunity to continue on our mission to transform the lives of heart failure patients through the power of gene therapy. Building on our success in Danon Disease to date, I am particularly excited to expand our cardiology focus and capabilities and address a clear unmet medical need in BAG3-associated dilated cardiomyopathy. By combining Renovacor’s compelling preclinical work with our joint clinical, regulatory and CMC expertise, we believe we will be well-positioned to bring the highest impact gene therapy with the best chance for success to these patients in the most productive and efficient manner possible.” Gaurav Shah, M.D., Chief Executive Officer of Rocket Dr. Shah continued, “Given the positive pediatric safety data previously announced from our Phase 1 RP-A501 Danon Disease program, and the upcoming pediatric efficacy data and longer-term adult cohort data we anticipate presenting at the Heart Failure Society of America (HFSA) Scientific Meeting at the end of this month, this strategic acquisition gives us what we believe is the broadest platform in the field to address these devastating rare cardiac diseases. Furthermore, the acquisition will bring to Rocket key personnel, namely a team of leading cardiology drug development experts, critical capabilities, and valuable IP to support continued development of the BAG3 as well as other potential cardiac programs, including a gene therapy research collaboration for arrhythmogenic cardiomyopathy.” Renovacor’s most advanced program, REN-001, is an AAV-based gene therapy targeting BAG3-associated dilated cardiomyopathy (DCM), a severe form of heart failure. BAG3-DCM represents a significant unmet medical need in a patient population with rapidly progressive cardiac dysfunction in whom no treatments targeting the underlying mechanism of disease exist. Renovacor has deep technical expertise in the development of precision therapies that address genetically driven cardiac diseases. Further, Renovacor is supported by world-class scientific collaborators, a robust intellectual property portfolio and personnel with expertise in BAG3-DCM. These assets and capabilities, all together, represent tremendous value and will enhance Rocket’s leading position in cardiac AAV-based gene therapy. “Renovacor has made tremendous progress in advancing targeted gene therapies to address the high unmet medical needs of patients living with genetically driven forms of heart disease,” said Magdalene Cook, MD, Chief Executive Officer of Renovacor. “Our experienced team is excited to join Rocket in a shared vision of broadening patient access to precision medicines for cardiovascular disease and addressing common barriers jointly. We look forward to combining the considerable resources and expertise of Renovacor and Rocket in creating a category leader in the precision cardiology field. As a result of this combination, we will be suspending current guidance regarding preclinical and clinical timelines for our programs as we evaluate these items with the Rocket team.” It is currently anticipated that the transaction will close by the first quarter of 2023, subject to approval by Renovacor and Rocket shareholders, receipt of any required customary regulatory approvals and the satisfaction of other customary closing conditions. RTW Investments, LP, a significant shareholder of both Rocket and Renovacor, has entered into a voting agreement with Renovacor, pursuant to which they have agreed, among other things, and subject to the terms and conditions of the agreement, to vote in favor of the Renovacor acquisition as a Rocket stockholder. SVB Securities is serving as exclusive financial advisor and Goodwin Procter LLP is serving as legal counsel to Rocket. Wells Fargo Securities is serving as exclusive financial advisor and Troutman Pepper Hamilton Sanders LLP is serving as legal counsel to Renovacor. About Rocket Pharmaceuticals, Inc. Rocket Pharmaceuticals, Inc. is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The Company’s platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rocket’s first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition. About Renovacor Renovacor is a biotechnology company focused on delivering innovative precision therapies to improve the lives of patients and families battling genetically-driven cardiovascular and mechanistically-related diseases. The company’s lead program in BAG3-associated dilated cardiomyopathy (DCM) uses gene transfer technology to address the monogenic cause of this severe form of heart failure. Renovacor’s vision is to bring life-changing therapies to patients living with serious genetic cardiovascular and related diseases, by developing medicines that target the underlying cause of disease and provide a transformative benefit and significant improvement to quality of life.

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