Exopharm

Exopharm is an Australian regenerative medicine biopharmaceutical company that is seeking to develop and commercialise exosomes as therapeutic agents – initially a product called Plexaris™ and later as a product called Exomeres™.

Australia-based Exopharm is a biotechnology company that develops exosome-based therapeutics for various diseases. The company has three technology platforms: LEAP, to purify exosomes at a commercial scale, EVPS, to target exomes and selected cells and tissues, and LOAD, to load RNA and DNA into exomes. Exopharm also uses its patented technology, LEAP, to isolate and purify exosomes from different sources, such as platelets and stem cells, and engineer them to deliver specific therapeutic molecules. The company is listed on the Australian Stock Exchange and trades under the ticker symbol “EX1.” As of August 2023, the company had not disclosed a clinical pipeline.

Novel therapies to reboot cells and organs

Cell & Gene Therapy

Tryp Therapeutics and Exopharm Limited Announce Completion of Plan of Arrangement

Australia-based Exopharm is a biotechnology company that develops exosome-based therapeutics for various diseases. The company has three technology platforms: LEAP, to purify exosomes at a commercial scale, EVPS, to target exomes and selected cells and tissues, and LOAD, to load RNA and DNA into exomes. Exopharm also uses its patented technology, LEAP, to isolate and purify exosomes from different sources, such as platelets and stem cells, and engineer them to deliver specific therapeutic molecules. The company is listed on the Australian Stock Exchange and trades under the ticker symbol “EX1.” As of August 2023, the company had not disclosed a clinical pipeline.Key customers and partnershipsExopharm partnered with biotechnology provider Sartorius BIA Separations in November 2021 to develop solutions for large-scale exosome production. Through the partnership, the companies would use the synergies between Sartorius BIA Separation’s Connective Interaction Media (CIM) monolith chromatography columns and Exopharm’s LEAP platform to discover solutions to technical limitations in large-scale and high efficiency exome production.Prior to this, in January 2021, Exopharm also entered an agreement with the subsidiary of biotechnology company Astellas Pharma Inc, Astellas Institute for Regenerative Medicine (AIRM), to manufacture exomes for Astellas. As a part of the signed agreement, Astellas would pay Exopharm up to USD 481,000 for the total project. Astellas will initially evaluate Exopharm’s technologies, and then contract Exopharm to load a specified amount of exomes with functional RNA and add specific molecules onto Astella’s surfaces (using Exopharm’s EVPS technology platform).

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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;&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. 
 
 
 As of May 2024, 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. 
 
 
 
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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 May 2024, 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 six gene therapeutics, one 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. 
 
 
 
 
 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 May 2024, 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;
 
 
 
 
 
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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 May 2024, the company had five therapeutic solutions being clinically tested. The company was developing four gene therapies for the treatment of retinitis pigmentosa, Leber congenital amaurosis, geographic atrophy, and orphan disease. Its treatment candidate for retinitis pigmentosa was in Phase III of clinical study, while its other gene therapies were in Phase I/II. 
 Ocugen was also developing a biological material, in the IND stage, for diabetic macular edema, diabetic retinopathy, and wet age-related macular degeneration. The company's cell therapy for the treatment of articular cartilage defects in the knee was in Phase III of clinical study. Finally, the company was also developing three vaccines, all in IND stages, for prevention of diseases caused by the flu, Covid-19, and a combination of the flu and Covid-19.
 
 
 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.
 
 
 
 
 
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Ocugen

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 MeiraGTx is a developer of gene therapies that aim to treat ocular diseases and problems, neurodegenerative disorders, and salivary gland and autoimmune disorders. MeriaGTx is also developing riboswitch-inducible expression programs for the treatment of inherited obesity and Wilson disease, along with producing small-molecule riboswitch inducers. The company is listed on the Nasdaq and trades under the symbol MGTX.&nbsp;&nbsp;
 
 
 As of May 2024, the company had four gene-based therapeutic candidates in Phase II of their clinical trials for the treatment of (1) xerostomia, Parkinson's Disease, and RPE65-Associated retinal dystrophy and (2) achromatopsia. The company also had one therapeutic being developed to treat Sjogren&rsquo;s Syndrome that was in Phase I/II of its clinical trials. Additionally, MeiraGTx had seven other therapeutics in discovery/preclinical stages. The company was also developing a therapeutic for X-linked retinitis pigmentosa in collaboration with Janssen, which was in Phase III of clinical trials. 
 
 
 Key customers and partnerships
 
 
 MeiraGTx partnered with the International Institute of Risk and Safety Management (IIRSM) in November 2022 to influence risk agendas, raise awareness, the standards of risk, and safety management across the industry.&nbsp;
 
 
 Funding and financials
 
 
 Meira last raised funds in October 2023, when the company secured a strategic investment of USD 30 million from Sanofi. As part of the transaction, Sanofi was granted the right of first negotiation for MeiraGTx's gene regulation technology in certain targets.&nbsp;
 
 
 
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MeiraGTx

Companies producing cells, genes, tissues, and biomaterials such as exosomes and extracellular matrix for drug developers and pharmaceutical companies developing regenerative medicine

Chimeric antigen receptor T-cell (CAR-T) therapy involves genetically engineering T cells to target and kill cancer cells

Companies conducting R&D on therapies that modify or introduce new genes into the body to treat diseases

Includes therapies that use cells (other than CAR-T and stem cells) such as Adipose-Derived Regenerative Cells (ADRCs), cardiosphere-derived cells, and gamma delta T cells (GDT cells)

Stem cell therapy involves using stem cells usually extracted from the bone marrow and umbilical cord blood to repair a variety of diseases

Startups in the hub consider individual patient profiles, involving variability in genes, environment, and lifestyle

Genomics helps understand the genetic information stored in the DNA to develop targeted treatments

Promising research and receding stigma are giving investors cannabis-boom flashbacks.

Psychedelic Medicine

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 The Canadian company Tryp Therapeutics has gained shareholder approval for a merger with Exopharm. The combined company will be operated under one entity. In <a href="https://tryptherapeutics.com/tryp-therapeutics-inc-announces-signing-of-definitive-agreement-with-exopharm-limited/"> December 2023 </a>, a formal agreement was reached for Exopharm to acquire all outstanding shares of Tryp Therapeutics. The projected completion date is May 1, 2024, providing all conditions are met.
 The merger will allow Tryp to continue its development of psilocin-based therapies. The company believes this move will enhance their visibility and attract a wider investor base.
 The newly united company will be known as "Tryptamine Therapeutics Limited". They plan to start trading on the Australian Securities Exchange under the ticker symbol "TYP" from May 15, 2024, subject to receiving necessary approvals and meeting certain criteria.
 
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Exopharm

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