Acorn Bioventures

X4 Pharmaceuticals is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, that specializes in developing novel therapeutics for primary immune deficiencies and cancer. Its lead drug candidate, mavorixafor, is an oral small molecule antagonist of the chemokine receptor CXCR4 and is currently undergoing various clinical trials, including a Phase III trial for patients with warts, hypogammaglobulinemia, infections, and myelokathexis syndrome, as well as trials for severe congenital neutropenia and clear cell renal cell carcinoma. The company is also advancing X4P-002, which targets glioblastoma multiforme, and X4P-003, aimed at treating primary immune deficiencies. Additionally, X4 Pharmaceuticals has a collaboration with Abbisko Therapeutics to explore mavorixafor in combination with checkpoint inhibitors for oncology applications. Founded in 2014, X4 Pharmaceuticals was previously known as Arsanis, Inc. before rebranding in 2019.

Neuron23, Inc. develops and manufactures medicines for genetic disorders using advanced artificial intelligence technology and genetics. Founded in 2018 and headquartered in South San Francisco, California, with an additional office in Munich, Germany, the company focuses on creating therapeutics for neurological diseases, including Parkinson's and Alzheimer's, as well as immunological disorders. By leveraging recent advancements in human genetics alongside its AI-enabled drug discovery and biomarker platforms, Neuron23 aims to provide innovative treatments for patients suffering from these debilitating conditions, enhancing their quality of life through targeted and effective therapies.

ONK Therapeutics Limited is focused on developing anti-cancer therapies that utilize natural killer cells to address unmet needs in treating blood cancers. Established in 2015 and based in Galway, Ireland, the company specializes in optimizing natural killer cells through advanced techniques such as the expression of chimeric antigen receptors, high-affinity CD16, and novel trail variants, along with the silencing of immune checkpoints. This innovative approach aims to enhance the efficacy of natural killer cells in targeting and eliminating tumor cells. Additionally, ONK Therapeutics collaborates with Avectas to create off-the-shelf cancer therapies, further broadening its impact in the field of oncology.

Renovacor, Inc. is a preclinical-stage biopharmaceutical company based in Philadelphia that specializes in developing gene therapies for cardiovascular diseases. Founded in 2013, Renovacor is focused on addressing dilated cardiomyopathy (DCM), a condition that affects over 3 million individuals in the United States. The company's lead program utilizes a recombinant adeno-associated virus (AAV) to create a gene therapy targeting patients with DCM caused by mutations in the Bcl2-associated athanogene 3 (BAG3) gene. This specific genetic mutation is estimated to affect around 35,000 individuals in the U.S. and a similar number in Europe, representing an orphan disease as defined by FDA guidelines. Patients with BAG3 mutations tend to be younger and experience a faster progression to advanced heart failure compared to those with ischemic heart disease. Currently, these patients receive standard heart failure treatments, but the five-year survival rate remains at only 50%. Renovacor aims to develop a BAG3 gene replacement therapy that could prevent disease progression in this vulnerable population.

Eliem Therapeutics, Inc. is a clinical-stage biotechnology company based in Boston, Massachusetts, specializing in the development of therapeutic drugs for hyperexcitability disorders. Founded in 2018, the company focuses on creating innovative analgesics aimed at addressing unmet medical needs in conditions such as chronic pain, psychiatric disorders, and epilepsy. Eliem's key drug candidate, ETX-155, targets major depressive disorder and focal onset seizures, reflecting the company's commitment to advancing neuroscience and translational medicine. Through its research and development efforts, Eliem Therapeutics aims to improve the quality of life for patients suffering from these challenging conditions.

OnKure, Inc. engages in the discovery and development of epigenetic therapies for the treatment of cancer. The company focuses on developing selective inhibitors of histone deacetylases. Its portfolio includes ONK102, an inhibitor that reduces the growth rate of tumors in hematological and solid tumor cancers. OnKure, Inc. was incorporated in 2011 and is based in Boulder, Colorado.

Aileron Therapeutics is a clinical-stage biopharmaceutical company dedicated to developing and commercializing a novel class of drugs known as stabilized cell-permeating alpha-helical peptides, particularly in the field of oncology. The company’s lead drug candidate, ALRN-6924, is currently undergoing multiple clinical trials, including Phase 2a studies for advanced solid tumors and peripheral T-cell lymphoma, as well as Phase 1 trials for acute myeloid leukemia and advanced myelodysplastic syndrome. Aileron is also working on a next-generation wild type p53 reactivator. Collaborations with notable institutions, such as Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and Pfizer, further enhance its research efforts, particularly in evaluating ALRN-6924 in combination therapies. Aileron Therapeutics was founded in 2001, originally named Renegade Therapeutics, and is headquartered in Watertown, Massachusetts.

Neuron23, Inc. develops and manufactures medicines for genetic disorders using advanced artificial intelligence technology and genetics. Founded in 2018 and headquartered in South San Francisco, California, with an additional office in Munich, Germany, the company focuses on creating therapeutics for neurological diseases, including Parkinson's and Alzheimer's, as well as immunological disorders. By leveraging recent advancements in human genetics alongside its AI-enabled drug discovery and biomarker platforms, Neuron23 aims to provide innovative treatments for patients suffering from these debilitating conditions, enhancing their quality of life through targeted and effective therapies.

Q32 Bio is a biotechnology company developing treatments for patients with severe autoimmune and inflammatory diseases. Q32 Bio is addressing dysregulation to help patients take back control of their lives in diseases with few to no treatment options, or those diseases where current therapeutics provide inadequate relief. Q32 Bio consists of a team of scientists and industry veterans with strong expertise in immunology. The team has a strong track record of discovering and developing new and innovative therapies.

Q32 Bio is a biotechnology company developing treatments for patients with severe autoimmune and inflammatory diseases. Q32 Bio is addressing dysregulation to help patients take back control of their lives in diseases with few to no treatment options, or those diseases where current therapeutics provide inadequate relief. Q32 Bio consists of a team of scientists and industry veterans with strong expertise in immunology. The team has a strong track record of discovering and developing new and innovative therapies.

Freeline Therapeutics is a clinical-stage biotechnology company focused on developing liver-directed gene therapies for bleeding disorders and other diseases. Based in Stevenage, United Kingdom, Freeline's lead product candidate, FLT180a, is undergoing Phase 1/2 clinical trials aimed at treating moderate to severe hemophilia B. The company's pipeline also includes FLT190, which is in dose-escalating Phase 1/2 trials for Fabry disease, FLT201 for type 1 Gaucher disease, and FLT210 for hemophilia A. Freeline utilizes a next-generation proprietary adeno-associated virus (AAV) vector platform, enhancing its potential for systemic gene therapy applications. Since its founding in 2015, the company has been dedicated to advancing innovative treatments in the field of gene therapy.

Cardiff Oncology is a clinical-stage oncology therapeutics company developing drugs that target cell division for the treatment of leukemias, lymphomas, and solid tumor cancers. It has a broad intellectual property and proprietary technology to measure circulating tumor DNA (ctDNA) in urine and blood to identify and quantify clinically actionable markers for predicting response to cancer therapies. The company offers its PCM technology at its CLIA/CAP-accredited laboratory and plans to continue to vertically integrate its PCM technology with precision cancer therapeutics. Cardiff Oncology (formerly known as Trovagene) was founded in 1999 and is headquartered in San Diego, California.