Methodology

All entries for: Investment

August 5, 2025

Lysoway Therapeutics

Investment

Lysoway Therapeutics raised $2.93 million to develop small-molecule modulators of lysosomal ion channels, including TRPML1 and TMEM175, to restore autophagic flux and enhance plasma membrane repair, enabling treatment of neurodegenerative and rare diseases caused by lysosomal dysfunction.

Disease Area: Rare Diseases
Source
Source
July 28, 2025

PAhR Therapeutics

Investment

PAhR Therapeutics raised $14 million to develop novel therapies for rare pulmonary arterial hypertension (PAH). As a clinical-stage company, it is advancing innovative molecular mechanisms to address unmet needs where conventional treatments fall short.

Disease Area: Rare Diseases
Source
Source
July 27, 2025

Eikonoklastes Therapeutics

Investment

Eikonoklastes Therapeutics raised $13.56 million to develop immunotherapies for rare and aggressive solid tumors, including triple-negative breast cancer, ovarian cancer, and metastatic melanoma.

Disease Area: Rare Diseases
Source
Source
July 24, 2025

Investment

Targepeutics raised $0.2 million to develop molecularly targeted therapies for rare cancers and other diseases, minimizing harm to healthy cells while treating difficult-to-treat conditions.

Disease Area: Rare Diseases
Source
Source
July 24, 2025

Juvena Therapeutics

Investment

Juvena Therapeutics raised $30.49 million to develop protein-based therapeutics that restore cellular function and promote tissue repair for chronic and rare diseases.

Disease Area: Rare Diseases
Source
Source
July 22, 2025

Avalyn Pharma

Investment

Avalyn Pharma raised $100 million to develop inhaled therapies for rare respiratory diseases, including idiopathic pulmonary fibrosis and interstitial lung diseases, improving efficacy while reducing side effects compared to oral treatments.

Disease Area: Rare Diseases
Source
Source
July 10, 2025

Acelot

Investment

Acelot Therapeutics received $8.7 million to develop a new treatment for ALS, targeting a key protein called TDP-43, which malfunctions in ALS, to slow or stop disease progression.

Disease Area: Rare Diseases
Source
Source
July 9, 2025

Transposon

Investment

Transposon Therapeutics received an undisclosed investment to develop nucleoside inhibitors for neurodegenerative and aging-related diseases such as progressive supranuclear palsy (PSP), amyotrophic lateral sclerosis (ALS), and frontotemporal dementia (FTD), all rare and debilitating neurological disorders.

Disease Area: Rare Diseases
Source
Source
July 8, 2025

Neutrolis

Investment

Neutrolis received $4.46 million to develop therapeutics that target neutrophils and reduce severe immune responses in acute and chronic inflammatory diseases through engineered enzyme analogs.

Disease Area: Rare Diseases
Source
Source
July 1, 2025

Syntis Bio

Investment

Syntis Bio raised an additional $5 million to continue developing late-stage preclinical oral therapies that leverage intestinal biology to treat metabolic and rare diseases through daily, nutrient-blocking formulations.

Disease Area: Rare Diseases
Source
Source
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