NellOne Therapeutics

NellOne Therapeutics is a regenerative medicine company that has been spun out of the U.S. Department of Energy's Oak Ridge National Laboratory in Oak Ridge, Tenn. Senior Staff Scientist Dr. Cymbeline Culiat in the Lab's Biosciences Division / Systems Genetics Group had identified the activity of the Nell1 gene's pathway in the formation of key mammalian tissues and organs, and Battelle Ventures founded NellOne to translate her basic science discovery into clinical applications. The company is dedicated to developing and commercializing Nell1 protein therapeutics for restoring tissue mass and function in patients recovering from heart attacks.

NellOne Therapeutics Inc. is a biotechnology company pioneering advances in regenerative medicine. The company's core technology is centered around the NELL1 signaling protein, which was discovered by its co-founder and president, Bem Culiat, during her research at Oak Ridge National Laboratory. The NELL1 protein plays a crucial role in fostering the growth and maturation of musculoskeletal and cardiovascular tissues in mammals, including humans.

Talk about regenerative medicine, potential drug for COVID-19 patients

Targeting complex diseases with complex molecules

Large-molecule Therapeutics

GRIDSMART Technologies Inc. Founder Bill Malkes Named CEO of NellOne Therapeutics

NellOne Therapeutics Inc. is a biotechnology company pioneering advances in regenerative medicine. The company's core technology is centered around the NELL1 signaling protein, which was discovered by its co-founder and president, Bem Culiat, during her research at Oak Ridge National Laboratory. The NELL1 protein plays a crucial role in fostering the growth and maturation of musculoskeletal and cardiovascular tissues in mammals, including humans.
NellOne Therapeutics leverages the NELL1 protein's healing properties to develop therapies that can restore injured tissue to its normal functional state. The NELL1 protein's mechanism of action stems from its activity during early mammalian development when it produces an extracellular matrix that promotes a pro-healing environment. This environment regulates inflammation, increases tissue survival under adverse conditions, recruits stem cells, and enhances blood vessel formation, ultimately facilitating the formation and maturation of new tissue to replace damaged tissues.
In 2020, the company pivoted its focus to develop NV1, a proprietary variant of the naturally occurring NELL1 protein, as a recombinant protein drug for healing lung tissue damaged by Covid-19 and other viral infections. Based on promising data from mouse and cultured human lung tissue studies, NellOne Therapeutics is pursuing a fast-track path toward obtaining approval for the new drug.

Innovent Biologics is a leading biopharmaceutical company founded in 2011 with the mission to develop and commercialize innovative and affordable medicines. The company focuses on oncology, cardiovascular and metabolic diseases, autoimmune disorders, and ophthalmology. As of March 2024, Innovent has ten approved products in the Chinese market, including TYVYT (sintilimab injection), BYVASDA (bevacizumab injection), SULINNO (adalimumab injection), and others. The company's core product, TYVYT, is an anti-PD-1 monoclonal antibody approved across seven indications, making it the most widely approved PD-1 inhibitor in China.
Innovent has a robust pipeline with three new drug applications under review, four assets in Phase III or pivotal trials, and 19 molecules in early clinical development. The company's innovative pipeline spans various modalities, including bispecific antibodies, antibody-drug conjugates (ADCs), and RNA therapeutics. In oncology, Innovent is advancing the next-generation immunotherapy combinations with ADCs, such as the CLDN18.2 ADC (IBI343) and the EGFR/B7H3 bispecific ADC (IBI3001). The company's metabolic pipeline includes mazdutide, a first-in-class GLP-1R/GCGR dual agonist in late-stage development for weight management.
Innovent has a strong emphasis on global innovation, with its Innovent Academy delivering eight novel molecules into IND-enabling stage in 2023. The company has established large-scale manufacturing facilities in Suzhou and Hangzhou to support global supply. Innovent collaborates with over 30 global healthcare leaders, reinforcing its commitment to advancing biopharmaceutical innovation worldwide.

Innovent Biologics

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 ADC Therapeutics is a clinical-stage biotechnology company focused on developing and commercializing next-generation antibody-drug conjugates (ADCs) for treating cancer. The company's ADCs leverage highly potent and targeted pyrrolobenzodiazepine (PBD) dimer technology, which causes the ADCs' antibodies to bind to specific tumor cell surface antigens. Once internalized, the potent PBD dimer is released inside the cell, creating covalent cross-links between the DNA strands, disrupting cell division without triggering DNA repair mechanisms.
 
 
 
 
 ADC Therapeutics' lead product candidate, Zynlonta, received FDA approval in April 2021 for treating adult patients with relapsed or refractory diffuse large B-cell lymphoma after two or more lines of systemic therapy. The company is also advancing a deep pipeline of other PBD-based ADCs targeting hematologic malignancies and solid tumors. As of May 2021, ADC Therapeutics reported revenues of USD 38.2 million, reflecting initial sales of Zynlonta.
 
 
 
 
 In March 2022, ADC Therapeutics acquired US commercial rights to Overrkey Biological's anti-HER2 ADC Overena, broadening its cancer treatment portfolio. The acquisition aligned with the company's strategic focus on expanding globally with its PBD-based ADC technology.
 
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ADC Therapeutics

Sirnaomics is a biopharmaceutical company engaged in the discovery and development of RNA therapeutics. The company focuses on innovative drugs for indications with unmet medical needs and large market opportunities. Sirnaomics utilizes two proprietary delivery platforms: the Polypeptide Nanoparticle Formulation (PNP) and the second generation of GalNAc conjugates called GalAhead.
Sirnaomics has established a robust pipeline of drug candidates, with several products in clinical and preclinical stages. The company's lead clinical product, STP705, has demonstrated positive results in Phase II clinical trials for the treatment of squamous cell carcinoma in situ (isSCC) and basal cell carcinoma (BCC). Additionally, STP705 is being explored for focal fat reduction in the medical aesthetics field. Another promising candidate, STP707, is a PNP formulation consisting of two siRNAs targeting TGF-β1 and COX-2 for intravenous treatment of solid tumors. STP707 has shown promising results in a Phase I basket clinical study, exhibiting stable disease response in 74% of evaluable patients.
Sirnaomics achieved a significant milestone by advancing STP122G, the first candidate utilizing the GalAhead technology, into clinical development. STP122G targets Factor XI for anticoagulation therapeutics and is currently undergoing Phase I clinical trials.
In March 2023, Sirnaomics' subsidiary, RNAimmune, received IND clearance from the FDA to initiate a Phase I clinical trial for RV-1770, an mRNA vaccine targeting respiratory syncytial virus (RSV). Additionally, RNAimmune obtained FDA clearance for a SARS-CoV-2 vaccine booster candidate, RV-1730.
Sirnaomics has established its manufacturing facility in China, enabling the transition from a biotech company to a biopharmaceutical corporation. The facility has successfully produced a full GMP batch of STP707 for human injection and is expected to support the company's clinical pipeline.
In July 2024, Sirnaomics announced the completion of IND-enabling studies for STP125G, an siRNA therapeutic targeting Apolipoprotein C3 (ApoC3) for the treatment of cardiovascular diseases. The positive results from non-human primate studies support the filing of an IND with the FDA.

Sirnaomics

Sesen Bio is a late-stage clinical company that previously focused on developing targeted fusion protein therapeutics for cancer treatment. Its lead product candidate, Vicineum, is a locally administered targeted fusion protein composed of an anti-epithelial cell adhesion molecule (EpCAM) antibody fragment tethered to a truncated form of Pseudomonas exotoxin A for the treatment of non-muscle invasive bladder cancer (NMIBC).
In February 2021, the FDA accepted Sesen Bio's Biologics License Application (BLA) for Vicineum to treat BCG-unresponsive NMIBC, granting Priority Review. However, in August 2021, the company received a Complete Response Letter from the FDA, requiring an additional Phase 3 clinical trial for potential resubmission. Following discussions with the FDA in March 2022, Sesen Bio aligned on most elements for the trial design and planned to meet again in mid-2022 to finalize outstanding items.
On July 15, 2022, Sesen Bio made the strategic decision to voluntarily pause further development of Vicineum in the US due to the incremental timeline and costs associated with the additional Phase 3 trial. The company shifted its primary focus to consummating a strategic transaction to maximize shareholder value while seeking a partner for Vicineum's further development.
In September 2022, Sesen Bio announced a pending merger with Carisma Therapeutics, a clinical-stage biopharmaceutical company developing immunotherapies based on engineered macrophages. This merger was approved by Sesen Bio's stockholders in March 2023, with the combined company operating under the name Carisma Therapeutics Inc. and trading on the Nasdaq under the ticker "CARM".
Upon closing the merger in March 2023, Sesen Bio declared a special cash dividend of USD 75 million (around USD 0.36 per share) to its stockholders and issued them Contingent Value Rights (CVRs) entitling them to potential proceeds from the sale of Vicineum and other assets, as well as a USD 30 million milestone payment under the Roche Asset Purchase Agreement.

Sesen Bio

ACELYRIN, INC. is a late-stage clinical biopharmaceutical company focused on developing transformative medicines for immunology and inflammatory diseases. The company's lead product candidate is izokibep, a next-generation inhibitor of interleukin-17A (IL-17A) designed to overcome limitations of monoclonal antibodies. Izokibep is being evaluated in multiple late-stage trials for moderate-to-severe hidradenitis suppurativa (HS), psoriatic arthritis (PsA), and noninfectious uveitis.
In March 2024, ACELYRIN reported positive top line data from a global Phase 2 billion/3 clinical trial of izokibep in PsA, meeting the primary endpoint of ACR50 at 16 weeks with high statistical significance. The trial also demonstrated robust clinical responses for higher hurdle endpoints like ACR70, PASI100, and composite measures, indicating izokibep's potential to provide clinically meaningful benefits. Additionally, ACELYRIN announced differentiated long-term 32-week data from a global Phase 2 billion trial of izokibep in HS, showing rapid, dose-ordered improvement across multiple disease manifestations, including HiSCR100 achieved earlier than reported by other IL-17 inhibitors.
ACELYRIN's other late-stage asset is lonigutamab, a subcutaneously delivered monoclonal antibody targeting IGF-1R being investigated for the treatment of thyroid eye disease (TED). In March 2024, ACELYRIN announced positive proof-of-concept data from a Phase 1/2 trial, demonstrating rapid improvements in proptosis and clinical activity score within three weeks after the first dose.
As of December 31, 2023, ACELYRIN had cash, cash equivalents, and short-term marketable securities totaling USD 721.3 million, providing a runway to support key late-stage data milestones and registrational studies across multiple indications. In January 2023, ACELYRIN acquired ValenzaBio, adding lonigutamab and other immunology assets to its pipeline.

ACELYRIN

Companies that develop engineered proteins that can target specific proteins or cells in the body

Companies that develop large molecules that can be used to target specific RNA molecules in the body, either to silence or replace them

Companies that develop large molecules that combine the targeting ability of monoclonal antibodies with the cytotoxic effects of chemotherapy drugs

Companies that develop large molecules that can be used to replace or supplement deficient or malfunctioning proteins in the body

Companies specializing in peptide-based treatments leverage short amino acid chains, known as peptides, to target and modulate various biological processes for therapeutic applications

AL/ML used to identify potential drug targets, and support protein engineering and optimization

NellOne Therapeutics

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