Piton Therapeutics

Piton Therapeutics is pioneering the development of life-changing therapeutics for chronic conditions with limited therapeutic options.  It employs a proprietary platform to discern the gut microbiome’s role in the cause and progression of disease. Therapeutics are developed to precisely target pathogenic pathways in the gut lumen. AI capabilities efficiently identify drug candidates with desired characteristics which are validated in the company’s lab. Products consist of small-molecule drugs. Piton does NOT develop live bacterial therapeutics (LBTs) or seek to modify the composition of microbiota in the gut.

Piton Therapeutics is a biopharmaceutical company focused on developing precision therapeutics for chronic and systemic diseases. The company's primary areas of focus include neurological, metabolic, and inflammatory conditions. Piton's approach to drug development is centered on exploiting the causal role of the gut microbiome in disease progression. By utilizing both large and small molecule drugs, Piton aims to accurately target mechanisms of action in the gut microbiome, representing an advancement in precision targeting and disease specificity for microbiome-focused therapeutics.

Precidiag, Inc.

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

Tissue Targeting Therapeutics

Precidiag

Piton Therapeutics is a biopharmaceutical company focused on developing precision therapeutics for chronic and systemic diseases. The company's primary areas of focus include neurological, metabolic, and inflammatory conditions. Piton's approach to drug development is centered on exploiting the causal role of the gut microbiome in disease progression. By utilizing both large and small molecule drugs, Piton aims to accurately target mechanisms of action in the gut microbiome, representing an advancement in precision targeting and disease specificity for microbiome-focused therapeutics.
The company employs sophisticated platform discovery capabilities to rapidly analyze the compositional and functional makeup of the microbiome. This information is used to create disease-specific, multidimensional ecological models of the gut microbiome, which are then examined to identify causal factors. Piton's drug discovery process is enhanced by a proprietary AI-enabled engine that facilitates efficient and effective selection of lead molecules. The company's therapeutics are designed to be orally dosed and gut-restricted, with the goal of minimizing side effects while potentially revolutionizing care for millions of patients.
Piton Therapeutics is actively advancing its pipeline. The company was conducting Investigational New Drug (IND)-enabling studies for precision, small molecule drugs targeting Ulcerative Colitis and Type two Diabetes. Additionally, Piton was optimizing a drug for Autism Spectrum Disorder and researching a causal hypothesis for Parkinson's Disease.

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 Generation Bio is a biotechnology company developing non-viral genetic medicines for rare and prevalent diseases. The company's proprietary platform combines immune-quiet DNA (iqDNA), a novel variant of closed-ended DNA, with a cell-targeted lipid nanoparticle (ctLNP) delivery system and a rapid enzymatic synthesis (RES) manufacturing process. This platform is designed to create gene therapies with drug-like properties, offering durable gene expression, the ability to re-dose for individual patient titration, and the potential to sustain effect over a lifetime. Generation Bio's approach aims to enable multi-year durability from a single dose, deliver large genetic payloads, and allow titration and redosing to adjust or extend expression levels in each patient. The company's pipeline includes programs for liver diseases such as hemophilia A, phenylketonuria (PKU), and Wilson's disease, as well as eye disorders like Stargardt disease and Leber congenital amaurosis type 10. In October 2023, Generation Bio announced the development of iqDNA, which demonstrated cytokine levels and tolerability comparable to chemically modified messenger RNA in mice and non-human primates. Generation Bio's RES manufacturing process has the potential to produce ceDNA at a scale of hundreds of millions of doses.
 
 
 
 
 Key customers and partnerships
 
 
 
 
 In March 2023, Generation Bio entered into a strategic partnership with Moderna to develop non-viral genetic medicines. The collaboration combines Moderna's expertise with Generation Bio's platform technologies to expand their respective non-viral genetic medicines pipelines. Under this partnership, Moderna is using Generation Bio's ctLNP delivery system and iqDNA technology to advance two immune cell programs and two liver programs. The deal included a USD 40 million upfront cash payment and a USD 36 million equity investment from Moderna. Generation Bio will also receive future development, regulatory, and commercial milestone payments, along with royalties on worldwide net sales of immune cell-targeted and liver-targeted products. The partnership aims to develop new lipid nanoparticles and expand in vivo immune cell targeting as a new class of genetic medicines.
 
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Generation Bio

Oncopeptides AB is a biotech company focused on developing therapies for difficult-to-treat hematological diseases, particularly multiple myeloma. The company's lead product, PEPAXTO (melphalan flufenamide, also known as melflufen), received FDA approval in February 2021 for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy. PEPAXTO is the first anticancer peptide-drug conjugate approved by the FDA for this indication. The drug works by leveraging aminopeptidases, which are overexpressed in multiple myeloma cells, to release cytotoxic agents.
In August 2022, the European Commission granted marketing authorization for Pepaxti (melphalan flufenamide) in combination with dexamethasone for the treatment of adult patients with multiple myeloma who have received at least three prior lines of therapies. Oncopeptides' research is based on two technology platforms: Peptide Drug Conjugates (PDC) and Small Polypeptide based Innate Killer Engagers (SPiKE). The PDC platform allows for efficient drug distribution in the body, enabling a wide therapeutic window and an optimized benefit-risk profile. The SPiKE platform utilizes the immune system to fight cancer by targeting natural killer cells, potentially reducing dose-limiting side effects while maintaining clinical efficacy.
As of 2023, Oncopeptides was conducting one clinical phase 3 study and six clinical phase 2 studies. The company's pipeline includes innovative drug candidates for difficult-to-treat hematological diseases, with the potential to expand into new indications. Oncopeptides' approach is science and data-driven, focusing on bringing innovation to patients with rare hematological diseases.

Oncopeptides

Neurona Therapeutics is a clinical-stage biotherapeutics company focused on developing regenerative cell therapy candidates for the treatment of neurological disorders. The company's lead investigational candidate, NRTX-1001, is being evaluated in an ongoing Phase I/II clinical trial for drug-resistant mesial temporal lobe epilepsy (MTLE). NRTX-1001 is a regenerative neural cell therapy derived from human pluripotent stem cells. It consists of fully-differentiated neural cells called interneurons that secrete the inhibitory neurotransmitter gamma-aminobutyric acid (GABA). Delivered as a one-time dose, these human interneurons are intended to integrate on-target and durably silence seizure activity in the epileptic region of the brain. In December 2023, Neurona presented early data from five participants in the ongoing study of NRTX-1001. Two patients who received the therapy more than a year prior experienced at least a 95% reduction in overall seizure frequency. NRTX-1001 is manufactured in Neurona's in-house GMP facility using proprietary methods. Beyond epilepsy, Neurona is also evaluating NRTX-1001 for potential application in Alzheimer's disease. The company's pipeline includes other cell therapy candidates such as investigational myelinating glial cells and gene-edited cells for undisclosed indications.

Neurona Therapeutics

Evox Therapeutics is a biotechnology company specializing in exosome therapeutics. The company utilizes its proprietary DeliverEX technology to engineer exosomes for drug delivery to specific organs, including the brain and central nervous system. Evox's approach aims to overcome limitations of genetic medicines such as gene therapy, gene editing, and RNA therapeutics by enabling safe, non-immunogenic repeated delivery to cells and tissues that are challenging to reach with other drug delivery technologies.
The company's technology focuses on harnessing and engineering the natural delivery capabilities of extracellular vesicles, known as exosomes, to develop a new class of therapeutics for treating severe rare diseases. Evox's intellectual property portfolio includes more than 100 granted patents across key jurisdictions, covering various aspects of exosome-based drug design, development, and manufacturing. In May 2023, Evox was granted a US patent for a method of purifying engineered exosomes using ultrafiltration and size-based separation steps, which is widely used in exosome therapeutics.
In June 2023, Evox acquired Codiak Biosciences' engEx-AAV technology platform, which enables active loading of adeno-associated virus (AAV) into exosomes. This technology has demonstrated improved AAV delivery, increased expression breadth in preclinical models, and shielding of AAVs from neutralizing antibodies. The acquisition aligns with Evox's strategy to enhance exosome-mediated delivery for genetic medicines such as gene therapy and genome editors.

Evox Therapeutics

Aro Biotherapeutics is a clinical-stage biotechnology company based in Philadelphia that specializes in developing tissue-targeted genetic medicines. The company's core technology is a proprietary protein platform called Centyrins, which enables efficient and tissue-specific delivery of siRNA and other genetic drugs. Aro's lead candidate, ABX1100, is being developed for the treatment of Pompe disease, a rare genetic disorder characterized by toxic glycogen accumulation in muscles. ABX1100 comprises a CD71 receptor-binding Centyrin conjugated to a small interfering RNA (siRNA) that targets the Gys1 gene, reducing levels and activity of the Gys1 enzyme in muscle tissues. The therapy has shown promising results in preclinical studies, demonstrating potent and muscle-specific reductions in Gys1 mRNA and protein in the Pompe mouse model. In October 2023, Aro initiated a Phase 1 clinical trial of ABX1100 in Canada, with initial data expected in 2024. The US Food and Drug Administration granted ABX1100 Orphan Drug Designation and Rare Pediatric Disease status. Beyond Pompe disease, Aro is exploring the potential of its Centyrin-siRNA conjugate programs for developing new treatments for autoimmune disorders.

Aro Biotherapeutics

Therapeutics that are designed to target specific cells to reach designated target sites

Therapeutics that are designed to target specific protein or protein receptors to reach designated target sites

Therapeutics that use different targeting approaches, such as bone-targeting or pressure-based, to help therapeutics identify specific target sites

Companies that offer contract research and manufacturing facilities that focus on tissue-targeting therapeutics for life sciences organizations

Precidiag

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