8VC seeks to enable industry transformation. In a wave of creative destruction, they believe emerging platforms will replace the decades-old technology infrastructure behind many industries, promoting greater innovation and prosperity.

DCVC Bio is a venture capital fund that invests in early-stage life science companies driven by deep-tech approaches. Advances in genetic sequencing, artificial intelligence, and automation are converging to transform existing life sciences technologies and speed the development of new ones. The firm is looking for companies affecting deep technological change in synthetic biology, medicine, and agriculture. DCVC Bio was founded in 2018 and is based in San Francisco, California, United States.

DCVC Bio

Ben Franklin has provided the Capital, Knowledge and Networks that help innovative enterprises compete in the global marketplace, generating wealth and supporting regional economic growth. Ben Franklin has invested more than $165 million to grow more than 1,750 regional enterprises, across all areas of technology. It has launched university/industry partnerships that accelerate scientific discoveries to commercialization. The Ben Franklin Technology Partners is an initiative of the Pennsylvania Department of Community and Economic Development and is funded by the Ben Franklin Technology Development Authority.

Ben Franklin Technology Partners

Since its start in 1855 as the nation's first hospital devoted exclusively to caring for children, The Children's Hospital of Philadelphia has been the birthplace for many dramatic firsts in pediatric medicine. The Hospital has fostered medical discoveries and innovations that have improved pediatric healthcare and saved countless children’s lives. Over 150 years of innovation and service to their patients, their families and their community, reflect an ongoing commitment to exceptional patient care.

The Children's Hospital of Philadelphia

Gaingels is a venture investment syndicate in service of the LGBT+ community and its allies, investing in companies with LGBT+ founders and C-suite leaders at all stages of growth, as well as in more established companies resolved on building more inclusive teams, with a global portfolio of 130+ companies and $70M in deployed investment capital to date.  The Gaingels network also actively supports its portfolio in identifying and recruiting diverse talent to their C-suite and board leadership teams and fosters a vibrant global community of industry leaders, investors, operators, and entrepreneurs who share a common goal for positive social change through business and successful investments.

Gaingels

Samsung Life Science Fund is an investment firm.

Samsung Life Science Fund

Modi Ventures is a Texas-based investment firm investing in fund managers and founders building companies shaping the future of humanity. The company's primary investment focus is into disruptive and emerging AI and Medical technology applications. Investments in successful funds include Khosla Ventures, Section 32, Artis Ventures, Draper Associates and Antler Global. Direct investments include, Starling Medicine and Cyberdontics. For more information please visit www.modivc.com.

Modi Ventures

Latus Bio

Latus Bio focuses on developing innovative gene therapy candidates using precision delivery to enhance the lives of patients. Latus identified proprietary AAV capsids for clinical use. The company plans to administer low doses of its product candidates, aiming to improve clinical safety and manufacturing.

<div>Latus Bio develops gene therapy solutions for central nervous system (CNS) disorders. Leveraging advanced adeno-associated virus (AAV) capsids, the company aims to enhance the precision and efficacy of gene delivery, which addresses the common challenges of traditional gene therapies, such as limited potency, off-target effects, and scalability issues. Its platform involves high-throughput screening of millions of novel capsid variants to identify those with the best tissue and cellular targeting capabilities, thereby minimizing potential immunogenicity and toxicity.</div>

Novel therapies to reboot cells and organs

Cell & Gene Therapy

Philly daily roundup: Tech walks; Join us at Builders; Latus Bio raises $54M

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 Latus Bio develops gene therapy solutions for central nervous system (CNS) disorders. Leveraging advanced adeno-associated virus (AAV) capsids, the company aims to enhance the precision and efficacy of gene delivery, which addresses the common challenges of traditional gene therapies, such as limited potency, off-target effects, and scalability issues. Its platform involves high-throughput screening of millions of novel capsid variants to identify those with the best tissue and cellular targeting capabilities, thereby minimizing potential immunogenicity and toxicity.
 
 
 The company&rsquo;s pipeline includes six gene therapy candidates, each designed for specific CNS targets such as Parkinson&rsquo;s Disease, Huntington&rsquo;s Disease, and Alzheimer&rsquo;s Disease​.&nbsp;
 
 
 Funding and financials
 
 
 In May 2024, Latus Bio secured USD 54 million in a 
 <a href="https://sp-edge.com/updates/29118">
 Series A
 </a>
 funding round led by 8VC and DCVC Bio, with participation from Samsung Life Science Fund and other undisclosed investors. The funding was intended for use in expediting the development of its gene therapies for CLN2 disease and Huntington&rsquo;s disease.&nbsp;
 
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<div>
 
 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

<div>
 
 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).
 
 
 
</div>

Sarepta Therapeutics

<div>
 
 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;
 
 
 
 
 
</div>

Mesoblast

<div>
 
 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.
 
 
 
 
 
</div>

Ocugen

<div>
 
 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

<div>
 
 Latus Bio Inc., a gene therapy company, has raised USD 54 million in a Series A funding round. The round was led by 8VC and DCVC Bio, and saw participation from the Samsung Life Science Fund and several other undisclosed investors. Samsung Life Science Fund is supported by Samsung Biologics Co., Samsung Bioepis Co., and Samsung C&amp;T Corp.
 The funding will be used by Latus Bio to accelerate the development of their gene therapies for CLN2 disease and Huntington’s disease. By 2025, the company aims to initiate human trials for the therapy designed for CLN2 disease.
 Based in Philadelphia, Latus Bio creates gene therapy solutions for central nervous system (CNS) disorders. The company uses advanced adeno-associated virus (AAV) capsids to increase the accuracy and effectiveness of gene delivery, addressing common challenges associated with traditional gene therapies such as limited potency, off-target effects, and scalability issues. The company's platform involves a high-throughput screening process for millions of novel capsid variants, enabling them to identify those with the best tissue and cellular targeting capabilities, thus reducing potential immunogenicity and toxicity.
 
</div>

Latus Bio

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