Capricor Therapeutics

Capricor Therapeutics is a clinical-stage biotechnology company that focuses on the discovery, development, and commercialization of cell and exosome-based therapeutics for the treatment and prevention of diseases.  Its  CAP-1002 is an allogeneic cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy and COVID-19.  The company was founded in 2005 and is headquartered in Los Angeles, California.

<div>Capricor Therapeutics is a listed biotechnology company that specializes in detecting, developing and marketing first-in-class cell and exosome-based therapeutics. This research and development will be used in the treatment and prevention of illnesses such as Duchenne muscular dystrophy (DMD), which is a genetic disorder that causes progressive muscular degeneration and affects many individuals in North America and Europe. Its main therapeutic technology is known as CAP-1002, which comprises cardiosphere-derived cells. Caprico is also developing COVID-19 vaccinations through its exsome therapeutic technology.</div>

Novel therapies to reboot cells and organs

Cell & Gene Therapy

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 Capricor Therapeutics is a listed biotechnology company that specializes in detecting, developing and marketing first-in-class cell and exosome-based therapeutics. This research and development will be used in the treatment and prevention of illnesses such as Duchenne muscular dystrophy (DMD), which is a genetic disorder that causes progressive muscular degeneration and affects many individuals in North America and Europe. Its main therapeutic technology is known as CAP-1002, which comprises cardiosphere-derived cells. Caprico is also developing COVID-19 vaccinations through its exsome therapeutic technology.
 
 
 
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 Tenaya Therapeutics is a developer of therapeutics for heart disease. The company develops its therapeutics using precision medicine, gene therapy, and cellular regeneration. Tenaya produces therapeutics that also use proprietary in-vivo and in-vitro models. Additionally, the company has developed in-house adeno-associated virus (AAV) capsid engineering capabilities and has screened over one billion variants from more than 30 diverse, proprietary AAV libraries. The company is publicly listed on the Nasdaq and trades under the ticker symbol TNYA.&nbsp;
 
 
 As of August 2023, the company&rsquo;s clinical pipeline included two candidates in Phase I clinical trials, a gene therapeutic for the treatment of Hypertrophic Cardiomyopathy, and a small molecule inhibitor for the treatment of heart failure with preserved ejection fraction. Additionally, Tenaya had one gene therapy candidate and two genetic medicine candidates in preclinical testing phases, along with five genetic medicine candidates in the discovery stage.&nbsp;
 
 
 In October 2023 Tenaya Therapeutics (Tenaya) received FDA clearance for the company&rsquo;s investigational new drug application (TN-401) to initiate clinical testing. TN-401 is Tenaya&rsquo;s adeno-associated virus serotype 9-based investigational gene therapy drug candidate for the treatment of arrhythmogenic right ventricular cardiomyopathy caused by mutations in the plakophilin-2 gene. 
 
 
 Funding and financials&nbsp;
 
 
 Tenaya&rsquo;s most recent funding raise was in November 2022, when the company secured around USD 75 million from gross proceeds from a public offering. The company did not disclose what the funds would be used for.&nbsp;&nbsp;
 
 
 In February 2024, Tenaya Therapeutics announced the pricing of its underwritten offering of 8,888,890 shares of its common stocks at an offering price of USD 4.50 per share. The company expects to receive total gross proceeds of approximately USD 50 million through the offering. 
 
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Tenaya Therapeutics

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 Sarepta Therapeutics is a gene therapy provider for conditions such as Duchenne Muscular Dystrophy (DMD), Limb-Girdle Muscular Dystrophy 
 (LGMD), and Charcot-Marie-Tooth Disease. The company&rsquo;s therapeutics incorporate technology related to gene therapy, RNA technologies, and gene editing. The company leverages a proprietary phosphorodiamidate morpholino oligomer (PMO) platform for the development of its RNA-based therapeutics. As of August 2023, the company had four FDA-approved treatments for DMD: ELEVIDYS, EXONDYS 51, VYONDYS 53, and AMONDYS 45. Sarepta Therapeutics is also listed on the Nasdaq, trading under the symbol SRPT.&nbsp;&nbsp;&nbsp;&nbsp;
 
 
 
 
 As of August 2023, the company had several RNA, gene, and gene editing therapeutics in various clinical stages. Sarepta had two RNA therapeutics, both for the treatment of DMD, one in clinical testing, and the other in pre-clinical testing. Additionally, the company had seven gene therapeutics, two for the treatment of DMD (both in clinical testing), four for the treatment of LGMD (three in clinical testing, one in preclinical testing) and one for multiple targets (in preclinical testing). Also, Sarepta had two gene editing-based therapeutics in development for the treatment of DMD, both in preclinical testing phases.&nbsp;
 
 
 
 
 Key customers and partnerships
 
 
 
 
 Over the years since its founding, Sarepta has partnered with several industry and academic partners. The list includes Dyno Therapeutics, Genethon, Genevant Sciences, Hansa Biopharma, Roche, Columbia University, Harvard University, Translational Laboratory of Murdoch University, University of Florida College of Medicine, The Abigail Wexner Research Institute at Nationwide Children&rsquo;s Hospital, University of Massachusetts Medical School, and the University of Western Australia.&nbsp;&nbsp;
 
 
 
 
 In October 2021, Sarepta and healthcare giant Roche announced they would collaborate on carrying out a clinical study of Sarepta&rsquo;s therapeutic candidate SRP-9001 for the treatment of DMD.&nbsp;
 
 
 
 
 Sarepta and pharmaceutical company Catalent announced that in January 2023, the companies were signing a commercial supply agreement. Through the agreement,&nbsp; Catalent would manufacture Sarepta&rsquo;s gene therapeutic for the treatment of DMD; delandistrogene moxeparvovec (SRP-9001).
 
 
 
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Sarepta Therapeutics

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 Mesoblast is a cell therapy manufacturer that provides therapeutics for oncology, cardiovascular, orthopedic, hematology, immunity, and inflammation-related conditions. The company is listed on the Australian Stock Exchange and trades under the ticker symbol &ldquo;MSB.&rdquo; Mesoblast develops therapeutics using its proprietary mesenchymal lineage cell technology platform, which is used to produce cryopreserved, off-the-shelf, and cellular-based solutions. The company also incorporates 3D bioreactor technology and automation into its production process to produce higher yields.&nbsp;
 
 
 
 
 As of August 2023, the company had five candidates that are in various stages of clinical testing. Mesoblast&rsquo;s steroid-refractory candidate for Acute Graft Versus Host Disease is currently in late-stage Phase III clinical testing and is the closest candidate to commercialization. The company&rsquo;s drug candidates for acute respiratory distress syndrome, chronic low back pain, and chronic heart failure and reduced ejection fraction are also in Phase III clinical testing while its candidate for inflammatory bowel disease is in Phase II of clinical testing.&nbsp;
 
 
 
 
 Key customers and partnerships
 
 
 
 
 The company has secured several agreements with international companies with regard to development and commercialization of Mesoblast&rsquo;s candidates, including Gr&uuml;nenthal GmBH, JCR Pharmaceuticals, Tasly Pharmaceutical Group, TiGenix NV, and the Lonza Group.&nbsp;
 
 
 
 
 Additionally, in April 2020, Mesoblast partnered with The US NIH&rsquo;s National Heart, Lung, and Blood Institute to conduct testing of its allogeneic mesenchymal stem cell (MSC) product candidate, &ldquo;remestemcel-L.&rdquo;&nbsp;&nbsp;&nbsp;&nbsp;
 
 
 
 
 Funding and financials
 
 
 
 
 The company raised USD 40 million in April 2023 in a global private placement, which saw participation from Mesoblast&rsquo;s existing major US, UK, and Australian shareholders. The company set aside the raised funds to launch and commercialize its lead candidate, remestemcel-L, in the treatment of children with steroid-refractory acute graft versus host disease, continue manufacturing of remestemcel-L at the Lonza Biosciences facility in Singapore, and initiative patient selection and enrolment its Phase III clinical trials of its rexlemestrocel-L candidate for low back pain associated with degenerative disc disease. 
 
 
 
 
 
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Mesoblast

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 Ocugen is a gene therapy manufacturer that develops vaccine, cell, and gene-based therapeutics for infectious, eye, and orthopedic diseases. Its cell therapy includes a three dimensional tissue-engineered cartilage disk through the growth of chondrocytes &ndash; the cells responsible for maintaining cartilage health. Its gene therapy solution consists of creating a copy of the patient&rsquo;s gene in order to replace mutated/damaged genes. The company is publicly listed on the Nasdaq, and trades under the symbol &lsquo;OCGN&rsquo;.&nbsp;
 
 
 As of August 2023, the company had five therapeutic solutions being clinically tested. Its inhaled vaccine candidate for the treatment of Covid-19 was in Phase III of clinical testing, along with its gene therapy solution for orthopedic diseases. The company also had three cell therapy-based therapeutic candidates two of which were in the Investigational New Drug-enabling stage, while its candidate for retinal degradation was in Phase I/II of clinical testing.&nbsp;
 
 
 Key customers and partnerships
 
 
 Ocugen partnered with biopharmaceutical manufacturer Jubilant HollisterStier of Spokane in June 2021 to manufacture its Covid-19 therapeutic COVAXIN. Through the partnership, Jubilant would be the potential commercial manufacturer of Ocugen&rsquo;s vaccine for the US and Canadian markets.
 
 
 
 
 Funding and financials
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 Ocugen last secured funds in May 2023 when the company raised USD 16.5 million through a public offering of common stock. Ocugen set aside the raised funds to use them for general corporate purposes, capital expenditures, working capital, and general and administrative expenses.&nbsp; 
 
 
 
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Ocugen

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 Athersys is a cell therapy manufacturer that provides stem cell-based therapeutics for neurological, inflammatory and immune, and cardiovascular-related diseases. The company is publicly listed on the Nasdaq under the symbol &ldquo;ATHX.&rdquo; The company&rsquo;s stem cell-based solutions fall under the brand name &ldquo;MultiStem,&rdquo; where the therapeutic is introduced intravenously for the downregulation of pro-inflammatory processes and upregulation of reparative immune responses to promote recovery.&nbsp;
 
 
 As of August 2023, the company had seven drug candidates in its clinical pipeline. Three are for the treatment of Ischemic Strokes, with all three candidates being either in Phase II or higher. One candidate, being developed for the treatment of trauma, is in the process of patient enrollment as a part of Phase II. Finally, Athersys&rsquo; final three candidates are being developed to treat Acute Respiratory Distress Syndrome (ARDS), and all candidates are in Phase II or higher stages of their clinical trials. The company additionally is developing cell-based therapies for animal health.&nbsp;
 
 
 Key customers and partnerships
 
 
 As of August 2023, the company was co-developing two drug candidates, one for the treatment of Ischemic Strokes and for the treatment of ARDS, with Japanese biotechnology provider Healios K.K. to be used exclusively in Japan. The companies originally partnered in August 2021, where Healios was additionally tasked with obtaining regulatory approval for the use of Athersys&rsquo; MultiStem solutions in Japan.&nbsp;
 
 
 Funding and financials
 
 
 The company last raised funds in April 2023, when it secured USD 3.7 million in a registered direct offering and a concurrent private placement to purchase and then sell ~3.7 million shares valued at USD 1 per share with warrants being exercised at USD 0.96 per share. The company did not disclose what the funds would be used for.&nbsp;
 
 
 
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Athersys Inc

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Capricor Therapeutics

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