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Leveraging personal data for custom-fit diagnosis and treatment

Precision Medicine

Jnana Therapeutics is a clinical-stage biotechnology startup company that focuses on the discovery and development of difficult-to-drug targets, using its next-gen RAPID chemoproteomics platform. The platform is used to discover small molecule treatments from highly validated targets. This platform uses a combination of chemistry, biology, and computational methods to identify and develop new drugs. The company's main focus is on therapeutic areas such as rare diseases, immune-mediated diseases, and cancer. Its lead drug candidate, JNT-517, is a small molecule drug targeting the SLC6A19 gene found in patients with phenylketonuria PKU, a rare genetic metabolic disease. The drug obtained the Rare Pediatric Disease Designation from FDA and has successfully dosed the first patient in its Phase I clinical trial. In January 2023, the FDA cleared Jnana’s first Investigational New Drug (IND) filing. Also as of June 2024, the company had four other targets in the discovery stage and additional .partnered undisclosed programs with Roche and Neurocrine Biosciences.Key customers and partnershipsThe company has two partnerships with pharma giant Roche. The two companies first collaborated in June 2020 with a focus on targeting SLC metabolite transporters. In November 2022, Jnana announced another collaboration involving the discovery and development of small molecule therapies targeting patients with cancer as well as immune-mediated and neurological diseases. The company was to receive USD 50 million as an upfront payment, with the potential to earn future payments exceeding USD 2 billion.

Ribon Therapeutics is a biotechnology company focused on developing therapeutics that target novel enzyme families activated under cellular stress conditions contributing to disease. As of January 2023, Ribon’s portfolio included two oral, potentially first-in-class clinical programs: RBN-2397, a PARP7 inhibitor, and RBN-3143, a PARP14 inhibitor, with broad potential applications in oncology and inflammatory diseases, respectively. The company aims to develop effective treatments for patients with limited therapeutic options by exploring novel areas of biology.Utilizing its proprietary BEACON+ (Blocking the Enzyme Activity Component of NAD+) platform, Ribon is creating a pipeline of selective, small molecule inhibitors for numerous NAD+-utilizing enzymes, starting with monoARTs (mono-ADP-ribosyltransferase), which have applications across various therapeutic areas.

Quanta Therapeutics is a biopharmaceutical company that focuses on developing targeted therapies for RAS-driven cancers. The company uses its proprietary Second Harmonic Generation platform and medicinal chemistry to develop oral small molecule drugs targeting KRAS mutations. Its approach targets multiple conformations of KRAS that were previously undruggable, expanding the scope of treatable cancers while evading resistance mechanisms.As of June 2024, the company's pipeline included three allosteric KRAS inhibitors: 1) QTX3034, a multi-KRAS inhibitor, 2) QTX3046, a G12D-selective KRAS inhibitor, and 3) QTX3544, a G12V-preferring multi-KRAS program. The company was also working on a novel RAF1 discovery program and undisclosed combination partners.

Therapeutics that target specific areas/tissues to minimize treatment side effects.

Tissue Targeting Therapeutics

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 Carisma Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative immunotherapies using its proprietary macrophage and monocyte cell engineering platform. The company's primary focus is on creating cell therapies to treat solid tumors and other serious diseases. Carisma's lead product candidate is CT-0525, a chimeric antigen receptor-monocyte (CAR-Monocyte) therapy targeting HER2-overexpressing solid tumors. This therapy is currently being evaluated in a Phase 1 clinical trial. CT-0525 is designed to address challenges in treating solid tumors with cell therapies, including tumor infiltration and immunosuppression within the tumor microenvironment. The company believes that CT-0525 has the potential for increased anti-tumor activity compared to its previous CAR-Macrophage therapy, CT-0508.
 
 
 
 
 In addition to its oncology pipeline, Carisma is exploring the use of engineered macrophages in treating fibrotic diseases. The company presented preclinical proof-of-concept data for its engineered macrophages in liver fibrosis at the American Society of Gene and Cell Therapy 2024 Annual Meeting. Carisma expects to nominate a development candidate for its liver fibrosis program in the first quarter of 2025.
 
 
 
 
 Carisma Therapeutics was formed through a merger with Sesen Bio in March 2023. The company is headquartered in Philadelphia, Pennsylvania. As of March 31, 2024, Carisma reported cash and cash equivalents of USD 56.5 million, which it expects will fund operations into the third quarter of 2025.
 
 
 
 
 Key customers and partnerships
 
 
 
 
 
 Carisma Therapeutics has established strategic partnerships with major pharmaceutical companies to advance its cell therapy platform. In January 2022, Moderna entered into a research collaboration with Carisma, paying USD 45 million to start the partnership. Additionally, Carisma has collaborated with Merck &amp; Co., with a trial expected to test Merck's immunotherapy Keytruda alongside Carisma's HER2-targeting treatment.
 
 
 
 
 In March 2022, Carisma announced a manufacturing agreement with Novartis, wherein Novartis would produce Carisma's HER2-targeted CAR-Macrophage cell therapy at its Morris Plains, New Jersey facility. This partnership demonstrates Novartis' entry into the contract development and manufacturing organization (CDMO) space for cell therapies.
 
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Kojin Therapeutics is a biotechnology company founded in 2019 and based in Boston, Massachusetts. The company is pioneering a new class of therapies based on cell state and ferroptosis biology. Ferroptosis is a recently discovered form of programmed cell death characterized by iron-dependent accumulation of oxidatively damaged phospholipids within cell membranes. Kojin is developing two classes of drugs: one that induces ferroptosis to kill cancer cells and another that blocks ferroptosis to prevent neurodegenerative diseases. The company's drug discovery platform integrates computational chemistry, structural biology, chemical biology, target-informed screening, and mechanism-driven pharmacology to modulate ferroptosis specifically.
Kojin's approach utilizes a novel non-genetic classification of cell states instead of conventional cell types to determine how cells respond to their environment or specific drugs. The company's proprietary molecular fingerprinting machine learning model connects complex cell states to biochemical processes like ferroptosis to identify targets for therapeutic intervention. This allows for the development of selective therapies tailored to specific diseases and tissues. The company had about 15 employees working in Boston's Seaport district as of January 2024.
Key customers and partnerships
Kojin Therapeutics' founding team includes prominent scientists such as Stuart Schreiber from Harvard University, Benjamin Cravatt from The Scripps Research Institute, and Stephanie Dougan from Harvard Medical School and Dana-Farber Cancer Institute. The company's scientific advisory board comprises experts in fields like oncology, chemical biology, and drug discovery.
Kojin has attracted investment from a syndicate that includes Cathay Health, Polaris Partners, Newpath Partners, Leaps by Bayer, AbbVie Ventures, Dana-Farber's Binney Street Capital, Eventide Life Sciences Fund, and Alexandria Venture Investments. In June 2021, the company raised USD 60 million in Series A funding to accelerate its drug discovery platform and advance its pipeline of therapeutics. While initially focused on oncology, Kojin's approach has potential applications across multiple disease areas including cardiovascular, immunologic, and degenerative diseases.

Reinventing gene therapies with next-generation technologies

Human Gene Editing

Caribou Biosciences specializes in gene editing to develop novel therapeutics for hematologic oncology indications using the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology. It develops allogeneic off-the-shelf gene edited therapies based on Chimeric Antigen Receptors T (CAR-T) cells and CAR Natural Killer (CAR-NK) cells.The company leverages its proprietary platform CRISPR hybrid RNA-DNA (chRDNA), which uses hybrid guides—both ribonucleic acid (RNA) and DNA nucleotides—to edit DNA. As of March 2024, Caribou had four candidates in its clinical pipeline, of which its lead candidate CB-010, a CAR-T therapy to treat relapsed/refractory B cell non-Hodgkin lymphoma, was in the Phase I clinical trial stage (March 2024). Its other CAR-T candidates, CB-011 for the treatment of relapsed/refractory Multiple Myeloma and CB-012, for the treatment of relapsed/refractory Acute Myeloid Leukemia, were also in Phase I of clinical trials. Its alternate application for CB-010 to treat autoimmune diseases was still in the discovery stage.The company intended to extend ex vivo (gene editing done in a specialized laboratory and returned to the body) therapies toward other indications outside oncology and implement its Cas12a chRDNA platform in in vivo (gene editing done inside the body) applications.Key customers and partnershipsThe company entered into a partnership with big pharma company <a href="https://news.abbvie.com/news/press-releases/abbvie-and-caribou-biosciences-announce-collaboration-and-license-agreement-for-car-t-cell-products.htm">Abbvie </a>in February 2021 for licensing and co-development of two CAR-T therapies. Caribou also entered into licensing agreements with <a href="https://www.businesswire.com/news/home/20201118005128/en/Caribou-Biosciences-Announces-Licensing-Agreement-for-scFvs-Targeting-CD371-to-Enable-Development-of-Allogeneic-Cell-Therapies">Memorial Sloan Kettering Cancer Center </a>(MSKCC) (November 2020), Corteva Agriscience, and Intellia Therapeutics. It also has a sales and assignment agreement with ProMab Biotechnologies Inc. (February 2020).Funding and financialsCaribou Biosciences went public on the Nasdaq Global Select Market in July 2021 and raised USD 304 million. The IPO proceeds were earmarked to fuel the company’s clinical development. For 2021, it recorded revenue of USD 9.6 million earned through licensing and collaborations.The company last raised funding in July 2023, when it raised USD 125 million in an upsized underwritten public offering. Caribou did not disclose what the funds would be used for.

Longevity Tech

Oisín Biotechnologies is a drug development company focusing on age-related diseases. When cells detect irreversibly damage, they enter a condition known as “cell-cycle arrest” or “senescence,” in which they are unable to kill themselves or resume normal function. Senescent cells accumulate with age, leading to a vast number of age-related diseases. Oisín is developing a patent-pending, DNA-targeted intervention to clear these senescent cells. A recent study has shown that clearing senescent cells reduces negative effects of aging pathologies and extends median lifespan. The company claims to have demonstrated the ability to transduce cells both in vitro and in vivo and that p16-positive senescent cells can be killed on demand in both environments. The company’s proprietary platform, SENSOlytics, is claimed to enable precise targeting of a senescent cell based on the DNA expression of the cell. By providing an exogenous apoptotic gene, which is only transiently expressed in cells that already have the p16 gene active, it is reportedly possible to precisely induce the senescent cell to commit suicide. The company claims to have shown as much as an 80% reduction in senescent cells in cell culture and significant reductions of senescent cell burden in naturally aged mice. Funds and financials In May 2021, Oisín Biotechnologies raised USD 5 million in a seed round led by Althea Groups. The funds were to be used in its work on chronic kidney disease.

RNA Therapeutics

Accent Therapeutics is a biopharmaceutical company focused on developing small molecule precision cancer therapies. The company targets critical intracellular dependencies that span multiple types of cancer, with a particular emphasis on RNA-modifying proteins (RMPs) and adjacent high-value areas. Accent's lead program is a first-in-class DHX9 inhibitor, which aims to address high unmet need indications including BRCA loss of function cancers (breast, ovarian), mismatch repair deficient (dMMR) or microsatellite instability-high (MSI-H) cancers (colorectal, endometrial, gastric), and other cancer types. DHX9 is a DNA/RNA helicase involved in various cellular processes, and its inhibition exploits key tumor vulnerabilities.
The company's second lead program is a potentially best-in-class KIF18A inhibitor. KIF18A is a mitotic kinesin motor protein critical for cell division in tumors with chromosomal instability. This inhibitor could potentially benefit patients with ovarian cancer and triple negative breast cancer (TNBC). Accent planned to initiate IND-enabling studies for KIF18A in the first half of 2024. In addition to these programs, Accent is developing inhibitors for other targets such as XRN1, ADAR1, and DHX9, which have shown promise in preclinical studies for various cancer indications.

Oisin Biotechnologies is a platform drug development company focused on creating drugs to combat a variety of age-related diseases. It is developing a highly precise, patent-pending, DNA-targeted intervention to clear these cells. As a recent study has shown, clearing senescent cells both reduces the negative effects of aging pathologies and also extends the median lifespan and survival. Oisin Biotechnologies was founded in 2014 and is headquartered in Seattle, Washington.

Third Arc Bio is a biotech company developing multifunctional antibodies for inflammatory and immunology diseases. Its drug development engine delivers superior biologics optimized to create  T-cell engagement by leveraging two technologies that power a pipeline of high-value therapeutics – a solid tumor synergy platform and an I&I platform that offers a novel approach to precision immune regulation at a tissue-specific level to achieve superior efficacy and safety.

The BaseLaunch accelerator supports carefully selected ventures over the course of 15 months and runs in two phases. It provides non-dilutive funding, no strings attached access to its Healthcare Partners, knowledge from highly experienced advisors, as well as a state-of-the-art infrastructure. Furthermore, Baselaunch embeds the ventures deeply within the Basel region, one of Europe’s finest biotech ecosystem. Baselaunch is backed by five industry leaders — Johnson & Johnson Innovation, Novartis Venture Fund, Pfizer, Roche and Roivant Sciences — as well as by other public and private partners.

Accent Therapeutics is a biopharmaceutical company developing oncology-focused, small molecule therapies in the emerging field of epitranscriptomics. This field of biology encompasses post-transcriptional chemical modifications of RNA that provide cells with a unique mechanism for regulating proteins critical for cellular growth and differentiation. By targeting cancer-linked RNA-modifying proteins (RMPs) with precision therapies, they aim to translate extraordinary science into life-changing therapies for patients.

DISCO Pharmaceuticals is a firm that specializes in large-scale surfaceome unlocking of cancer cells.

Mozart Therapeutics is a biotech startup focusing on the development of disease-modifying therapies for autoimmune and inflammatory diseases. The company is using a novel regulatory CD8 T cell network to create disease-modifying CD8 Treg modulators for a variety of autoimmune diseases. Mozart is developing a pipeline of first-in-class 'CD8 Treg modulators with the goal of restoring long-term immune balance and preventing the progressive damage caused by an autoreactive and pathogenic immune response.

Quanta is a biotechnology company uncovering novel cancer therapeutic candidates to target driver oncogenes. It develops allosteric modulation to directly restore conformational control of active RAS signaling at the cell membrane to inhibit the full scope of RAS-driven cancer types.

Capsida is a biotechnology company that specializes in tackling monogenetic and sporadic CNS and non-CNS disorders. It features a proprietary adeno-associated virus (AAV) engineering platform that generates capsids optimized to target specific tissues and cells in the diseased organ.

Jnana Therapeutics is a biotechnology company focused on discovering and developing medicines that improve patient lives. Utilizing its RAPID platform, the firm accelerates the identification of therapeutic targets and the creation of small molecule therapies for various diseases. Jnana aims to enhance drug discovery efficiency through advanced chemoproteomics techniques, addressing unmet medical needs.

Carisma Therapeutics is a biotech company that develops novel chimeric antigen receptor macrophage technology to treat solid tumors. Its cell therapies apply powerful chimeric antigen receptor technology and adoptive cell transfer in cancer therapy and to macrophages that infiltrate the solid tumor micro-environment that enables solid tumor patients to receive strong cellular immunotherapy and cancer treatments.

AmbAgon Therapeutics is a developer of cancer molecule therapeutics. The company offers research of a small molecule that augments a tumor-suppressing protein. AmbAgon Therapeutics was founded by Christian Ottmann, Lucas Brunsveld, and Michelle Arkin.

Nitrase Therapeutics is a platform company developing drugs against a newly identified class of enzymes called Nitrases, initially targeting Parkinson’s disease. The therapies that Nitrome is developing will target these enzymes and potentially help slow or halt the progression of diseases such as Parkinson’s. While the initial focus is on Parkinson's disease, the company aims to expand its proprietary platform to include other disease indications.

Community Hope is helping individuals, including veterans, and their families, overcome mental illness, addiction, homelessness, and poverty by providing housing and support services. Homeless veterans suffering from mental health issues and combat-induced trauma have been able to access their residential programs and support services.

For more than 157 years, Eskenazi Health has offered high-quality, cost-effective and patient-focused health care to the residents of Marion County and Central Indiana. As one of America’s largest essential health-care systems, Eskenazi Health provides treatment and services to nearly 1 million outpatient visitors each year.

Roanoke Chowan Community Health Center offers behavioral health, pharmacy, telehealth, clinical and pediatric care services.

The Night Ministry is a nonprofit organization that provides housing, health care, and social services.

Delaware Valley Community Health operates as a non-profit firm, the firm offers adult & family medicine, pediatrics, dental, etc. services.

Health Brigade is a non-profit organization that provides health services. Their services include wellness, health outreach, advocacy, needle exchange, and eligibility for services. They provide adult general medicine, including medical services for diabetes, high blood pressure, asthma, men’s health, psychiatric evaluation and medication, routine cancer screening, and chronic heart disease.

Good Samaritan Health Center provides medical, dental, behavioral, nutrition education, and wellness services.

Aaron E. Henry Community Health Services Center offers primary and preventive health care services.

MLK Health Center provides general health care services. They offer services in routine physical exams, prescribing necessary medications, treatment of minor illnesses and injuries, managing chronic conditions, screening for common health issues, and management of acute health conditions. They also offer a thorough assessment of patient data, disease data, and medicine-related data where patient-specific medicine-related problems are identified, including ineffective dosages, non-adherence, and adverse drug reactions.

The Institute for Family Health is offering mental health, dental care, and social services.

Starday Foods is a healthy and sustainable food products company.

Stemson Therapeutics is driven by a vision where anyone battling the emotional trauma or social stigma of hair loss has an opportunity to truly cure their condition and safely restore their natural hair. Our engineered cells are combined with a unique tissue engineering solution and are capable of generating a net new supply of hair follicles for patients in need. Stemson

Ribon Therapeutics is a biotechnology company pioneering the discovery of new cancer medicines that target monoPARP proteins, critical regulators of cancer survival mechanisms. The diverse and central functions that PARPs, or poly(ADP-ribose) polymerases, play in cancer cell survival pathways are just emerging, and many of the seminal discoveries in the field have been made by Ribon’s scientific founders. By combining insights from our founders and an experienced management team, Ribon is building a proprietary drug discovery platform to investigate the molecular action and biological function of a subset of the PARP protein family, called monoPARPs. Based on its platform, Ribon is creating a pipeline of innovative medicines that selectively target monoPARPs to treat cancer and may have the potential to impact other diseases. The company is backed by prominent life science investors, including The Column Group, U.S. Venture Partners, Deerfield Management and Osage University Partners.

Founded by Stuart Schreiber, Benjamin Cravatt, Stephanie Dougan, and Vasanthi Viswanathan, Kojin is accelerating a drug discovery platform that connects complex cell states to known biochemical processes such as ferroptosis, or iron-dependent cell death, and enables the development of selective therapies for a broad range of hard-to-treat diseases.  The company’s investors include Polaris Partners, Newpath Partners, Cathay Health, Leaps by Bayer, AbbVie Inc., Eventide Asset Management, Alexandria, and the Dana-Farber Cancer Institute venture firm, Binney Street Capital.

Caribou Biosciences is an operator of a medical technology company intended to analyze cell and genome engineering. The company specializes in cellular engineering and analysis based on the CRISPR-Cas9 technology platform which paired with a guide RNA, cuts double-stranded DNA and allows for specific changes to DNA, enabling the development of therapeutics for agricultural biotechnology, industrial biotechnology, and basic and applied biological research.

Faze Medicines is a biotechnology company that uses a variety of screening and proteomics techniques to define condensate interaction networks.

Year Up's mission is to close the Opportunity Divide by providing urban young adults with the skills, experience, and support that will empower them to reach their potential through professional careers and higher education.

Palleon Pharmaceuticals is a developer of a biotechnology platform designed to offer glycoimmune checkpoint inhibitors to treat cancer.The company's biotechnology platform integrates technologies and insights from scientific leaders all over the world in the fields of glycoscience and human immunology to create a novel approach to treating cancer by targeting multiple immune cell types, providing physicians with a wider range of rational combination therapies to treat cancer and tackle resistance to first-generation immuno-oncology agents.

Enthera is a new Italian biotech start-up whose mission is to find new therapeutic approaches to treat diabetes and related gastrointestinal complication (enteropathy), as well as other intestinal disorders sharing the same biological pathway. Its name comes from the crasis of the terms entero (referring to the intestine) and therapy.   Enthera was founded by BiovelocITA in October 2016 in collaboration with the scientists: Prof. Paolo Fiorina and Dr. Francesca D’Addio.

Jacobio Pharmaceuticals, founded in 2015 in Beijing China, is a clinical-stage biotech company dedicated to the development of global truly innovative drugs for the unmet needs in China and abroad. The vision and mission of Jacobio have become a global leader to provide revolutionary therapies for serious and life-threatening diseases such as cancer, autoimmune and infectious diseases. Jacobio is founded by seasoned executives from top global and local pharmaceuticals companies who have an astounding track record of success and backed by prestigious venture capital firms such as Lilly Asia Ventures and Qiming Capitals. Dr. Yinxiang Wang, the co-founder and chairman of Jacobio, also is one of the founders of Betta Pharmaceuticals, an A-share listed innovative pharmaceutical company, and the national ''The Recruitment Program of Global Experts''. The most senior management team has over 20 years of drug R&D experience and successfully brought several innovative drugs into the clinical and market stage. Within 3 years, Jacobio has quickly established a compelling drug R&D team of over 100 employees and one-stop capability covering target discovery, med-chemistry, pharmacology, CMC formulation, clinical, and registration. With those, the company has built up a rich pipeline of 8-10 assets with clear competitive advantages and excellent drug properties. JAB-3068, the most advanced program for Jacobio, has focused on a novel target potential for both immunotherapy and target therapy and enter Phase I clinical trial in US and IND approved at China and clinical trials would be initiate at China very soon.

Quench Bio is a developer of gasdermin biology drugs designed to treat severe inflammatory diseases.The company leverages insights into gasdermin biology and innate immunity to develop medicines with lead compounds that target and inhibit the pore-forming protein that mediates the release of inflammatory cytokines, alarmins, DNA and NETs, enabling patients to get relief from inflammatory diseases.

Pact Pharma is a developer of personalized adoptive T cell therapies designed for the eradication of solid tumors.The company's therapies help in the identification of neo-epitopes that serve as private mutations for each patient's cancer as well as creates an opportunity to engineer autologous T cells that target and kill tumors expressing these neo-antigens, enabling physicians to recognize and remove each patient's cancer cells.

Inventiva Pharma is a clinical-stage biopharmaceutical company focused on the development of oral small molecule therapies for the treatment of patients with significant unmet medical needs in the areas of fibrosis, lysosomal storage disorders and oncology. They have built a pipeline backed by a discovery engine with an extensive library of proprietary molecules, a wholly-owned research and development facility and a team with significant expertise and deep experience in the development of compounds that target nuclear receptors, transcription factors and epigenetic modulation. Leveraging these assets and expertise, they are advancing two clinical candidates in three indications, as well as a deep pipeline of earlier stage programs.

Prevail Therapeutics is a biotechnology company focused on developing novel gene therapies for patients with Parkinson’s disease and other neurodegenerative diseases. Prevail was launched in 2017 by The Silverstein Foundation for Parkinson’s with GBA, REGENXBIO, and OrbiMed, and is headquartered in New York, NY.

St. Jude Children's Research Hospital® was founded in 1962 by philanthropist and performer Danny Thomas. It is the first and only National Cancer Institute-designated Comprehensive Cancer Center dedicated solely to children. Since its opening, St. Jude has strived to advance cures for children with cancer and other catastrophic diseases through research and treatment.  As one of the largest pediatric oncology centers in the world, St. Jude Children’s Research Hospital has treated children from all 50 states and around the world. Today, St. Jude has one of the world's leading pediatric brain tumor programs, as well as one of the largest bone marrow transplant and sickle cell disease programs in the United States.  Headquartered in Memphis, Tennessee, St. Jude has eight affiliate clinics across the United States. The hospital also shares its breakthroughs with resources like St. Jude Cure4Kids and St. Jude Cloud, which enable doctors and scientists across the globe to access St. Jude findings, helping them save even more lives. Aligned with Danny Thomas’ vision, St. Jude is committed to ensuring no child is denied treatment based on race, religion, or financial standing. This is largely made possible by the American Lebanese Syrian Associated Charities® (ALSAC®), the hospital’s awareness and fundraising organization. ALSAC focuses on connecting with donors and spreading awareness about the organization while St. Jude focuses on improving survival rates through innovative, cutting-edge research and technology. Major goals of this research include understanding the molecular, genetic and chemical bases of catastrophic diseases in children, as well as finding new life-saving treatments and methods of prevention for said diseases. To learn more about St. Jude Children’s Research Hospital and its lifesaving mission, visit https://www.stjude.org.

Rheostat’s mission is to invent novel treatments for neurodegenerative disease through modulation of mitophagy and autophagy. They believe that the degradation of toxic cellular components, whether they are damaged organelles or aggregated proteins, is a fundamental node of biology.  Mutations that impair these clearance pathways cause multiple neurodegenerative diseases, many of which are associated with cognitive impairment. They aim to leverage our understanding of these critical pathways to discover and develop novel small molecules that will restore cellular balance and treat neurodegenerative and rare diseases.

Ribometrix is a platform therapeutics company that discovers small molecule drugs that target functional 3D RNA structures to treat human diseases. Our scientific offices are located in the newly renovated Biolabs Space in Durham, with offices in Chapel Hill and Boston.

Paragen Bio is a provider of a technology intended to treat autoimmune diseases. The company's technology helps in treating diseases which causes the immune system to attack healthy tissues and organs, enabling doctors to stop the deterioration and complete destruction of tissue.

Magnolia Neurosciences aims to discover and develop proprietary, selective, and drug-like small molecule therapeutics for the prevention of neuronal cell death, thereby providing novel treatment options for patients suffering from neurodegeneration and related conditions. Magnolia Neurosciences Corporation, created to pursue technologies developed at The University of Texas MD Anderson Cancer Center’s Therapeutics Discovery Division and the Neurodegeneration Consortium (NDC), is a New York-based Accelerator Life Science Partner portfolio company.

Artios is an independent DNA Damage Response (DDR) company focused on developing first-in-class treatments for cancer. Established in May 2016, the Company is led by an experienced scientific and leadership team with proven expertise in DDR drug discovery. Artios is building a pipeline of next-generation DDR programs, including through a unique partnership with Cancer Research Technology (CRT), the development and commercialization arm of Cancer Research UK (CRUK), and with leading DNA repair researchers worldwide.  The Company’s investors include SV Life Sciences, Merck Ventures, Imperial Innovations, Arix Bioscience PLC, CRT Pioneer Fund (managed by Sixth Element Capital), and AbbVie Ventures. Artios is based at the Babraham Institute in Cambridge, UK.

Alector is combining state-of-the-art antibody technology and recent discoveries in neuroimmunology and human genetics to develop novel therapeutics for Alzheimer’s disease, other forms of dementia, and mechanistically related neurodegenerative disorders. Alector’s strategy is to efficiently generate and validate antibody drugs with unique functional properties that engage key disease-altering targets. This approach is enabled by a strategic alliance with Adimab, the industry leader in discovery and optimization of antibody therapeutics. Alector is currently developing leads for 4 major targets and anticipates taking 2 of these through pre-clinical development and IND enabling studies within 24 months. Alector has incorporated a highly integrated and lean biotechnology structure with extensive outsourcing that supports core scientific and management teams.

Quentis Therapeutics is a developer of immuno-therapeutics designed to address ER stress in multiple diseases. The company's therapeutics are designed to target ER stress pathways and awaken the immune system's ability to fight cancer, enabling patients to get access to new treatments for cancer.

Exicure is Formerly Known as AuraSense Therapeuticsis. Exicure is developing a new class of immunomodulatory and gene silencing drugs against validated targets. Our 3-dimensional, spherical nucleic acid (SNA™) architecture unlocks the potential of nucleic acid therapeutics in multiple organs. Our lead programs address diseases from inflammatory disorders to oncology.  SNA constructs overcome one of the most difficult obstacles to nucleic acid therapeutics: safe and effective delivery into cells and tissues. SNA constructs exhibit unparalleled transfection efficiency into numerous cell and tissue types including the skin without carriers or transfection agents. Moreover, SNAs can be used as potent immunotherapeutic agents for the treatment of cancer or infectious disease.

Disarm Therapeutics is a biotechnology company that creates disease-modifying therapeutics for patients with axonal degeneration. Its drugs prevent the loss of axons and treat neurological diseases including multiple sclerosis, amyotrophic lateral sclerosis, glaucoma, and peripheral neuropathies that enable patients to avail treatment for acute diseases of the central, ocular, and peripheral nervous systems. Disarm Therapeutics was founded in 2016 and is headquartered in Cambridge, Massachusetts.

Rodeo Therapeutics is a drug development company focused on novel enzyme target and biological pathway that play a critical role in tissue repair and regeneration.  Rodeo’s initial goal is to develop novel small molecules therapies for the treatment of inflammatory bowel disease and the promotion of blood cell reconstitution following bone marrow transplant.

eFFECTOR is a clinical stage biopharmaceutical company focused on pioneering the development of a new class of oncology drugs referred to as selective translation regulator inhibitors (STRIs). eFFECTOR’s STRI product candidates target the eIF4F complex and its activating kinase, mitogen-activated protein kinase 1/2 (MNK 1/2). The eIF4F complex is a central node where two of the most frequently mutated signaling pathways in cancer, the PI3K-AKT and RAS-MEK pathways, converge to activate the translation of select messenger RNA into proteins that are frequent culprits in key disease driving processes. Each of eFFECTOR’s product candidates is designed to act on a single protein that drives the expression of multiple functionally related proteins, including oncoproteins and immunosuppressive proteins in T cells, that together control tumor growth, survival and immune evasion. eFFECTOR’s lead product candidate, tomivosertib, is a MNK 1/2 inhibitor.  KICKSTART, a randomized, double-blind, placebo controlled Phase 2b trial of tomivorsertib in NSCLC in combination with pembrolizumab is currently open for enrollment. Zotatifin, eFFECTOR’s inhibitor of eIF4A, is currently in the dose-escalation portion of a Phase 1/2 trial, with Phase 2a expansion cohorts expected to initiate in the second half of 2021. eFFECTOR has a global collaboration with Pfizer to develop inhibitors of a third target, eIF4E.

Aquinnah Pharmaceuticals is focused on an exciting new area of research and development to modulate neurodegenerative stress granules, believed to be important in the pathology of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.

ApoGen Biotechnologies, Inc. is focused on the development of new class of therapeutics targeting drivers of cancer genomic mutation.As the arsenal of available cancer therapies has grown and evolved from cytotoxic chemotherapy to targeted therapy to immunotherapy, one fact has unfortunately remained constant nearly all cancer drug therapies eventually fail due to the development of drug resistance. Mutation of cellular DNA is central to the formation of cancer, and chronic mutation of the cancer genome is a primary cause of cancer evolution and drug resistance, resulting in ineffective therapy, cancer recurrence and metastasis, and decreased overall survival.

Morphic Therapeutic is a biotechnology company that develops oral integrin therapies to treat serious chronic diseases, including autoimmune, cardiovascular, and metabolic diseases, fibrosis, and cancer. The company offers the development of therapies for patients with immunological, fibrotic, neoplastic, and vascular diseases, focusing on creating small molecule inhibitors of various integrin targets.

Lodo Therapeutics Corporation is a drug discovery and development company focused on the creation of naturally derived novel therapeutics that will have a dramatic impact human health on a global basis. Lodo seeks to work in partnership with global pharmaceutical companies and world leading Non-Governmental Organizations (NGO’s) to tackle some of the greatest challenges in human health:  resistant infectious disease and cancers.   Lodo Therapeutics was created to pursue the scientific vision of Dr. Sean Brady at Rockefeller University. Dr. Brady and his laboratory have developed a genome-based, culture-independent platform for the discovery, biosynthesis, and characterization of small molecules from microbial sources present in soil samples.  Lodo believes that the potential cures for a number of deadly and/or debilitating diseases literally lie at our feet. By combining the advancements in DNA sequencing and bioinformatics, this innovative discovery platform exploits the power of microbial evolution for the identification of therapeutically valuable pharmaceutical products derived from natural sources.   Lodo Therapeutics, an Accelerator Corporation-backed entity, is headquartered in New York City.   The company’s lab and offices are located in the Alexandria Center for Life Science, New York City’s first and only premier life science park.

Petra Pharma is a clinical-stage pharmaceutical company developing a diverse portfolio of novel therapies for serious medical conditions such as cancer and metabolic diseases. The company also focused on novel enzyme targets that play a central role in a variety of important cellular processes, including cell division, growth, trafficking, and signaling. It will develop novel small molecules for the treatment of cancer and metabolic diseases to target these cellular processes in order to therapeutically impact human health. Petra Pharma was founded in 2016 and is headquartered in New York, USA.

AM-Pharma B.V., a biopharmaceutical company, engages in the development and commercialization of therapeutics for human use in the Netherlands. It focuses in the pre-clinical and clinical development of novel therapeutics to treat inflammatory and infectious diseases. The company develops products based on endogenous proteins and protein derived peptides, as well as molecules that naturally occur in the human body. Its products include alkaline phosphatase for treatment of acute kidney injury and ulcerative colitis; and immune response amplifying peptide hLF1-11 for the treatment of drug resistant hospital acquired infections. The company was founded in 2000 and is headquartered in Bunnik, the Netherlands.

Virobay, Inc. operates as an anti-viral drug discovery and development company. It engages in developing, investigating, and creating treatments for hepatitis C, such as protease and polymerase inhibitors. The company was founded in 2006 and is headquartered in Menlo Park, California.

Kala Pharmaceuticals is developing novel treatments for diseases that affect mucosal tissues. The company's proprietary mucus penetrating particle technology dramatically improves drug distribution and pharmacokinetics by uniformly coating the mucosal surface with biocompatible, drug-loaded particles. Kala's formulations achieve high, sustained local drug concentrations with reduced systemic exposure and are delivered in a patient-friendly format. The company's research efforts have demonstrated successful in vivo proof-of-concept in mucosal organs such as the respiratory tract, eye, and cervicovaginal tract, enabling diverse product opportunities that target significant clinical unmet need. Kala is focused on advancing an internal pipeline of products through human proof-of-concept while establishing R&D collaborations with select partners to improve products in their pipelines.

Avaxia Biologics is a private, early-stage company developing orally-delivered antibody therapeutics using a proprietary antibody platform. The company is pursuing products for oral mucositis, inflammatory bowel disease, and diabetes, and obesity. Avaxia Biologics was founded in 2005 and headquartered in Lexington, Massachusetts.

AbbVie is a biopharmaceutical company that develops pharmaceuticals and medical devices. The company provides products and services to therapeutic areas including immunology, oncology, neuroscience, eye care, virology, women's health, and gastroenterology advancing treatments across various therapeutic areas.

<div> AbbVie Inc. (NYSE: ABBV) is a research-based biopharmaceutical company headquartered in North Chicago, Illinois. Founded in 2013 as a spin-off from Abbott Laboratories, AbbVie focuses on developing and commercializing advanced therapies for some of the world's most complex and critical conditions. The company reported revenues of USD 54.32 billion in 2023 and employs approximately 50,000 people globally. AbbVie's key therapeutic areas include: <ul> <li> Immunology: Products like Humira, Skyrizi, and Rinvoq for conditions such as rheumatoid arthritis, psoriasis, and Crohn's disease </li> </ul> <ul> <li> Oncology: Treatments including Imbruvica and Venclexta for various cancers </li> </ul> <ul> <li> Neuroscience: Medications such as Botox Therapeutic, Vraylar, and Ubrelvy for neurological and psychiatric disorders </li> </ul> <ul> <li> Aesthetics: Products like Botox Cosmetic and Juvederm for cosmetic applications </li> </ul> <ul> <li> Eye Care: Treatments including Lumigan and Restasis for various eye conditions </li> </ul> <ul> <li> Virology: Medications like Mavyret for hepatitis C </li> </ul> In 2024, AbbVie completed the acquisition of ImmunoGen, adding Elahere, a treatment for folate receptor-alpha positive platinum-resistant ovarian cancer, to its portfolio. The company continues to invest heavily in research and development, with an estimated R&amp;D spend of USD 6.50 billion in 2023. AbbVie has been recognized for its workplace culture, ranking on Fortune's 100 Best Companies to Work For list in 2024. The company has also increased its quarterly dividend for 11 consecutive years since its inception, demonstrating its commitment to returning value to shareholders. </div>

AbbVie's atogepant and onabotulinumtoxinA receive strong recommendations in the newly updated Canadian Headache Society (CHS) Migraine Prevention Guideline Français

AbbVie Biotech Ventures

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

Psychedelic Medicine

Companies conducting research on psychedelic compounds and developing novel therapeutics.

Companies operating digital platforms to deliver psychedelic therapies including psychedelic integration, virtual experiences, and mental coaching and tracking. AI drug discovery companies specializing in psychedelic therapeutics are also included.

Companies operating clinics and treatment centers to deliver psychedelic therapies.

Companies supplying psychedelic compounds through cultivation, chemical synthesis and biosynthesis for medical research and drug development.

Companies building a patent portfolio of novel analogs and derivatives of first-generation psychedelics and new drug delivery mechanisms. 

Companies providing genetic tests to identify individuals with high risk or at-risk of having hereditary disease, aid physicians with a diagnosis, and support clinical decision making.

Secure cloud-based platforms and data sharing networks that collect, index, store, and analyze large amounts of health data and patient population data sets from sources including genomic, clinical, wearables, imaging, and population data.

Biopharma and biotechnology companies that develop precision medicine drugs based on novel disease biomarkers present in multi-omic patient population data and genetic insights.

Companies that study intergenomic variations in genes associated with drug response and metabolism to match patients with the appropriate drug and dosage.

Companies that develop an in-vitro medical device or imaging tools and tests that identify genetic mutations for both diagnostic (nature of the disease) and prognostic (likely course of the disease) purposes, allowing providers to match a patient to a specific drug or therapy.

Software analytics platforms that integrate multiple omics datasets (genomics, transcriptomics, proteomics, and metabolomics) to gain insights and support target discovery, drug development, clinical trials, and diagnostics.

INCUMBENTS_PRESENT

<div>
 Pharmaceutical company AbbVie has purchased biotech firm Nimble Therapeutics for an upfront cost of USD 200 million, with further possible payments based on reaching specific milestones. The deal, AbbVie's third of this kind this year, intends to amplify the scope of their immunology pipeline via Nimble's expertise in developing oral peptide therapeutics and their high-throughput peptide synthesis and optimization technology. 
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