Monthly Archives: March 2022

"Throughout his career, Rayne has demonstrated strong leadership in multiple roles across the globe, including most recently as chief manufacturing officer leading the development of our state-of-the-art manufacturing facility in Thousand Oaks," said Peter Anastasiou, president and CEO of Capsida. "Rayne's appointment to COO will ensure Capsida achieves its goal of moving our technology from the lab and to patients, and will help Capsida realize the promise of gene therapy."

"I'm honored to take on the role of COO at Capsida, a company that always puts patients first," said Mr. Waller. "Throughout my career in the biotechnology industry, I've worked to focus on the end goal of bringing therapies to patients who have no other options. As COO, I look forward to working with the Capsida leadership team and our board to bring the promise of gene therapy to life."

Gene therapy is still in its infancy and has yet to achieve its full potential. Capsida's proprietary, targeted, non-invasive gene therapy technology allows more selective targeting of specific tissues and cells, overcoming many of the problems associated with first-generation gene therapies, specifically off-target cell and organ activity. In addition, it allows the gene therapy to be delivered non-invasively through intravenous administration.

Prior to joining Capsida, Mr. Waller spent 27 years at Amgen in increasing roles of responsibility across manufacturing and supply-chain management. His most recent roles at Amgen included vice president and site head for Amgen's largest manufacturing site in Puerto Rico, vice president of regional manufacturing, responsible for overseeing site operations for manufacturing facilities in Ireland and The Netherlands, and vice president of global supply-chain management with responsibility for contract manufacturing, global supply management, operations strategic planning, and risk management.

Mr. Waller holds a bachelor's degree in business administration from the University of Arizona.

About Capsida Biotherapeutics

Capsida Biotherapeutics Inc. is an industry-leading gene therapy platform company creating a new class of targeted, non-invasive gene therapies for patients with debilitating and life-threatening genetic disorders. Capsida's technology allows for the targeted penetration of cells and organs, while limiting collateral impact on non-targeted cells and organs, especially the liver. This technology allows for the delivery of the gene therapy in a non-invasive way through intravenous administration. Capsida's technology is protected by a growing intellectual property portfolio which includes more than 30 patent applications and one issued U.S. patent 11,149,256. The company is exploring using the technology across a broad range of rare and more common genetic disorders. Its initial pipeline consists of multiple neurologic disease programs. The company has strategic collaborations with AbbVie and CRISPR, which provide independent validation of Capsida's technology and capabilities. Capsida is a multi-functional and fully integrated biotechnology company with proprietary adeno-associated virus (AAV) engineering, multi-modality cargo development and optimization, translational biology, process development and state-of-the-art manufacturing, and broad clinical development experience. Capsida's biologically driven, high-throughput AAV engineering and cargo optimization platform originated from groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., a neuroscience professor at the California Institute of Technology. Visit us atwww.capsida.comto learn more.

SOURCE Capsida Biotherapeutics

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Capsida Biotherapeutics Announces Appointment of Rayne Waller to Chief Operating Officer - PR Newswire

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You don't go to the leading edge of life sciences/bioproduction looking for bargains, as the strong growth and margins available to companies facilitating the rapid growth of new biologic treatment options like gene and cell therapies have fueled strong share price performance (and multiples) for larger, better-known companies like Danaher (DHR) and Thermo Fisher (TMO), as well as smaller players like Bio-Techne (NASDAQ:TECH).

I find a lot to like in Bio-Techne's leverage to cell and gene therapy-enabling products like GMP protein production and non-viral gene editing, as well as its leverage to life sciences/bioproduction research tools, spatial biology, and molecular diagnostics. I don't find nearly as much to like in the valuation, but stocks like these are a "you either get it, or you don't" sort of proposition where you're basically betting that the underlying growth of the market and the company's strategic decisions will eventually lead to enough revenue and profit growth down the line to redeem an eye-watering valuation today.

Through both M&A and leveraging internal manufacturing capabilities grounded in a long history of producing proteomic research reagents, Bio-Techne has built a business segment to leverage the exceptional underlying growth potential in cell and gene therapies over the coming years.

Bio-Techne's offerings include Cloudz cell activation kits that use non-magnetic beads to separate and activate cells, the TcBuster non-viral gene editing platform, and its GMP proteins, as well as complementary tools in spatial biology and immunocytochemistry. All told, management has sized its opportunity in CGT at over $3B today, growing at over 20%, and the company only has a small low-single-digit market share today.

GMP proteins are a major thematic driver right now, growing 180% in the fourth quarter and helping drive 80%-plus growth overall for the Cell and Gene Therapy (or CGT) business. GMP proteins are proteins produced in dedicated facilities and in accordance with strict quality management guidelines, ensuring biological activity, purity, and batch-to-batch consistency.

These proteins are not used directly in therapies (they're not infused or injected into patients), but they're used to produce cell and gene therapies that are practically in their infancy. As such, I think a handy shortcut for readers is to think of them as consumables used in the development and manufacture of cell and gene therapies like Novartis' (NVS) Kymriah (CAR-T) and Zolgensma (gene therapy).

Management recently opened a new GMP protein manufacturing facility and the ramp of commercial operations there should drive meaningful growth for several quarters, with the business scaling from around $30M to over $200M over the next several years. In addition to ramping up this facility, management has already started talking of broadening the business into GMP antibodies and media, as well as RNA and DNA products. As an aside, Bio-Techne tried to acquire Aldevron, a manufacturer of GMP-grade plasmid DNA, mRNA, and proteins back in 2021 but lost out to Danaher.

Bio-Techne's CGT operations are the "sizzle" today, but there's plenty of steak behind it. While the company's legacy proteomic reagents and diagnostics (calibrators and controls) operations aren't particularly sexy, they are still growing at a mid-single-digit clip and they generate good cash flow for the business.

Beyond this are interesting opportunities in analytical instrumentation, spatial biology, and molecular diagnostics.

The Analytical Solutions business is growing at a double-digit rate for Bio-Techne, with the company leveraging growing demand for automation in the lab. The Simple Western is the only automated tool for western blotting, a technique that identifies specific proteins in a sample and can require hours of hands-on time. The Maurice platform automates protein profiling while Ella, a benchtop immunoassay platform, can be used in a range of applications like monitoring cytokine storms (in CAR-T recipients or COVID-19 patients) and detecting impurities in finished biologics production runs.

Bio-Techne's Advanced Cell Diagnostics business offers RNAscope and DNAscope, and an in-situ RNA/DNA hybridization platform that detects target RNA or DNA within a single cell without disturbing the tissue morphology. This allows users to examine biomarker status, structural variation, and so on, and is useful in a range of applications from cancer diagnostics to ensuring whether reprogrammed T-cells for CAR-T are expressing the intended antigen receptors.

In molecular diagnostics, Bio-Techne hasn't seen the hoped-for adoption of its ExoDx Prostate IntelliScore liquid biopsy for prostate cancer (a rule-out test after ambiguous PSA results), but reimbursement has improved and post-pandemic normalization of office visits should drive more use. Beyond this, though, the company is building out a broader portfolio of biomarker-based screening products that can address a range of diseases/conditions, with a particular focus on genetic carrier screening and oncology diagnostics.

All told, Bio-Techne is targeting markets with combined addressable revenue of over $15 billion a year, including high-growth areas like analytical research tools, cell/gene therapy, spatial biology, liquid biopsy, and molecular diagnostics. Given that cell and gene therapies are practically in their infancy (or at least early toddlerhood), I see significant growth opportunities in tools and consumables that facilitate research and production, and I think Bio-Techne has a good starting position today.

I also doubt that the company is anywhere close to done with its M&A program. More than a decade ago, I followed Bio-Techne (then Techne) as a sell-side analyst, and the change in the company since then has been remarkable. Much of that change can be tied to a change in management and operating philosophy (actively targeting growth), but M&A has played a key enabling role, including deals for Asuragen (diagnostics kitting), B-MoGen (TcBuster gene editing), Quad Technologies (Cloudz), Exosome, CyVek (Ella) and ProteinSimple (automated equipment, including Simple Western).

Most recently, the company announced an unusual deal for Wilson Wolf, the manufacturer of G-Rex bioreactors (used to grow/produce proteins or cells like T-cells in volume). Bio-Techne isn't buying the company today but now has the option to buy 20% when the company reaches $100M in revenue and the whole company at $225M in revenue (or $135M in EBITDA), as well as another option if those targets aren't reached. No definitive terms have been reached, but at $225M in revenue, it seems likely that the deal would cost over $2B.

I'm looking for high-teens annualized revenue growth over the next decade and EBITDA margin expansion to over 42% in 2024 (as well as long-term FCF margins in the high 20%s), but those targets don't really matter. I don't think there's any way to derive a bottoms-up fair value that will look attractive today, though comparative analysis to other high-growth life sciences/bioproduction names suggests a current EV/forward revenue of around 16.5x isn't completely unprecedented (Danaher paid 17.6x forward revenue for Aldevron).

Investing in highly-valued "hyper-growth" stocks has never really been in my wheelhouse; I've done it and made money doing it, but I'm always reluctant to make "forget the valuation and buy it'll all work out eventually" recommendations to others. I do like Bio-Techne's leverage to growth in bioproduction generally and cell/gene therapy research and production more specifically, but this is definitely not a stock for investors who can't accept high risk.

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Bio-Techne - Exceptional Leverage To Biopharma Growth, And Priced Accordingly - Seeking Alpha

LONDON--(BUSINESS WIRE)--Ixaka Ltd, an integrated cell and gene therapy company, today announces an expansion of its IP portfolio to allow a broad range of applications for its polymeric targeted nanoparticle (TNP) gene delivery platform across multiple therapeutic areas. The extended IP enables the development of therapies encapsulating any cargo including mRNA, plasmids and adenovirus associated virus (AAV), and gene editing technologies as well as lentiviral vector-based therapies.

Ixakas TNP composed of proprietary polymer capsule (OM-PBAE polymers (oligopeptide end-modified poly -amino-ester polymers)) directed to specific cells through targeting agents attached the capsule (an aptamer-based targeting moiety) and cell targeted efficient transduction (use of lentiviral vector cell specific promoter). This targeting enables the targeted nanoparticles to be directed to specific cells allowing beneficial gene transduction to occur directly within a patients body. The technology is currently being applied as a gene delivery platform to generate CAR T-cell therapies in vivo for haematological malignancies, with the potential for improved efficacy and safety compared to similar products currently marketed or in development. The expanded IP allows for a broad range of cargos to be encapsulated providing greater flexibility to engineer new therapies which are optimized to specific diseases.

A new agreement extends the use of its OM-PBAE polymers for encapsulation of a broad range of cargos for use in any therapeutic area including mRNA, plasmids, and any other vectors in addition to lentiviral vectors and AAV.

The extension of the license agreement opens the door to expansion of Ixakas TNP platform, enabling the use of OM-PBAE polymers capsules to be used for delivery of numerous cargos with the choice of genetic modification technology tailored for each disease. Potential applications include drug delivery for oligonucleotides such as DNA, RNA and siRNA, plasmids, small molecules, and gene editing using tools such as CRISPRCas9, zinc finger or megaTALS enabling Ixaka to broaden its therapeutic pipeline.

Joe Dupere, CEO at Ixaka, commented:

This IP agreement further strengthens Ixakas rapidly growing IP portfolio, highlighting the pioneering nature and broad potential of our targeted nanoparticle technology. The platform is already showing great potential in generating CAR-T cells in vivo for CD19 blood cancers. With an array of other possible applications, we will now be seeking collaborations for our future pipeline, which could encompass solid tumours, rare genetic disorders, autoimmune diseases, broader immunotherapy applications, gene editing, immunodiagnostics and vaccines.

IP portfolio overview

Building a robust and broad IP portfolio is at the heart of Ixakas development strategy. The Companys ongoing R&D and upcoming clinical data will allow filing of additional patent applications across multiple territories.

Ixakas current IP portfolio contains 1 patent family for its multi-cell therapy platform and 11 patent families for its TNP platform, covering all key components of the technology, including a proprietary polymer, bald engineered lentiviral vector, T-cell specific promoter and aptamer-based targeting agent. The portfolio also provides protection across a wide geographic range (including Europe, the US, Canada, Mexico, Brazil, China, India, Korea, Japan, Australia).

The patent is for products developed under a licence agreement between Ixaka (previously aratinga.bio), Sagetis Biotech (Sagetis) and universities (Institut Quimic de Sarria CETS Fundacio Privada and Institut dInvestigacions Biomediques).

About Ixaka

Ixaka is a cell and gene therapy company focused on using the natural powers of the body to cure disease.

Ixakas proprietary technologies enhance the naturally therapeutic power of cells by increasing the presence of curative cells at the site of disease, or by directly modifying cells within the body to improve disease targeting and boost their restorative effect.

Ixakas technologies concentrated multi-cell therapies and nanoparticle therapeutics demonstrate potential for the treatment of a broad range of serious diseases across oncology, cardiovascular, neurological and ocular diseases, and genetic disorders.

Ixaka has offices in London, UK with R&D and manufacturing operations in Seville, Spain and Paris, France and additional manufacturing capability in Frankfurt, Germany.

For more information, please visit http://www.ixaka.com

Connect with us: Twitter: https://twitter.com/Ixaka_Ltd ; LinkedIn: https://www.linkedin.com/company/ixaka-limited

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Ixaka Expands IP Portfolio to Enable Use of Targeted Nanoparticle in vivo Gene Delivery Technology With Any Cargo in Any Therapeutic Area - Business...

Wet age-related macular degeneration (wAMD) has recently witnessed a series of approvals thanks to a degree of product differentiation, and this has drawn enthusiasm from multiple stakeholders in the field. This interest is expected to be widened with the anticipated approvals of more products, particularly biosimilars. Despite this, a lack of treatment options for other sub-types of the disease means that opportunities are still available for pharmaceutical companies to make a difference to patients affected by AMD.

Product differentiation has been a key strategy employed by companies whose products have recently received approvals for wAMD. Consider for example Roches Susvimo (ranibizumab), which received US Food and Drug Administration (FDA) approval in October last year. Patients implanted with Susvimo are expected to visit their doctor only twice yearly to have the implant refilled so that the medicine can be delivered to their eyes continuously. That represents a significant alleviation of treatment burden when compared with the current standards of care such as Lucentis (ranibizumab), Eylea (aflibercept) and Beovu (brolucizumab), which need to be administered more frequently.

Vabysmo (faricimab), also from Roche, received FDA approval in January this year for wAMD and for diabetic macular oedema. This therapy is administered every four weeks for the first four doses, following which, depending on the results of optical coherence tomography and visual acuity evaluations, there is potential for the frequency of administration to be reduced to as few as twice a year, resulting in a lower cost of treatment compared to standard of care. While it remains to be seen to what extent these new therapies will be commercially successful, being associated with lesser frequency of administration already makes such therapies an attractive option for patients and physicians in this field.

Looking ahead, anticipated launches of new biosimilars and innovator molecules are expected to widen interest in the field of AMD. According to GlobalDatas Pharmaceutical Intelligence Centre (PIC), there are currently 15 products in late-stage development exclusively for wAMD in the seven major markets (7MM: the US, France, Germany, Italy, Spain, UK and Japan). Five of these are aflibercept biosimilars and four are ranibizumab biosimilars. While both groups of biosimilars are expected to directly target patient shares from their branded counterparts, Eylea (aflibercept) and Lucentis (ranibizumab), the latter two also face threats from innovator molecules including RGX-314 from RegenxBio and AbbVie. This is a gene therapy that has the potential to be used as a one-time treatment option for patients with wAMD and, if approved, has the potential to disrupt current market dynamics for this sub-type of AMD.

In addition, there are gaps that exist in the market, particularly for therapies that can help patients with dry AMD and geographic atrophy, for which there are currently no approved therapies available. According to GlobalDatas PIC, there are three therapies in late-stage development for geographic atrophy: Alkeus Pharmaceuticals ALK-001, Apellis Pharmaceuticals pegcetacoplan, and IVERIC bios avacincaptad pegol sodium. Dobecure SLs therapy ethamsylate is also in development for both dry AMD (dAMD) and wAMD. It remains to be seen, however, how effective these therapies will be in addressing the disease at an earlier stage, as it is crucial to treat these diseases before they progress further.

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Product approvals and unmet treatment needs expected to widen interest in AMD - Pharmaceutical Technology