New Enterprise Associates is a prominent venture capital firm focusing on investments in technology and healthcare startups. They help entrepreneurs build transformational businesses across multiple stages, sectors, and geographies. With over $19 billion in cumulative committed capital since the firm’s founding in 1977, NEA invests in technology and healthcare companies at all stages in a company’s lifecycle, from the seed stage through the IPO. The firm's long track record of successful investing includes more than 210 portfolio company IPOs and more than 360 acquisitions.

New Enterprise Associates

MPM Capital is a healthcare investment firm that invests in companies developing therapeutic technologies in oncology. The company invests in life sciences companies that seek to cure diseases by translating science into clinical outcomes. The company prefers to invest in the biotechnology, healthcare, and life sciences sectors.

MPM Capital

ATP, a global leader in life sciences venture capital, is developing the next generation of transformative biotechnology companies. ATP creates science-based companies starting with assets at various stages: from working with scientists on pre-IP ideas, to spinouts from existing company pipelines. The firm provides flexible capital, strategic insight, and operational resources to build sustainable, research-driven enterprises. ATP invests in its portfolio companies from seed stage through IPO and beyond and supports their management teams in strategic transactions and synergistic business combinations.

Apple Tree Partners

Cerecor

Cerecor is a biopharmaceutical company focused on becoming a leader in the development and commercialization of treatments for immunologic, immuno-oncologic and rare genetic disorders. The company is advancing its clinical-stage pipeline of innovative therapies that address unmet patient needs within rare and orphan diseases. The company's rare disease pipeline includes CERC-801, CERC-802 and CERC-803, which are in development for congenital disorders of glycosylation and CERC-006, an oral mTORc1/c2 inhibitor in development for the treatment of complex lymphatic malformations. The company is also developing two monoclonal antibodies, CERC-002, and CERC-007. CERC-002 targets the cytokine LIGHT (TNFSF14) and is in clinical development for treatment of severe pediatric-onset Crohn's disease and COVID-19 acute respiratory distress syndrome. CERC-007 targets the cytokine IL-18 and is in clinical development for the treatment of Still’s disease (adult onset Still’s disease (AOSD) and systemic juvenile idiopathic arthritis (sJIA)) and multiple myeloma (MM). CERC-006, 801, 802 and 803 have all received Orphan Drug Designation and Rare Pediatric Disease Designation, which makes all four eligible for a priority review voucher upon FDA approval.

Cerecor Inc., formerly known as Avalo Therapeutics, is a publicly-traded biotechnology company headquartered in Rockville, Maryland. The company focuses on developing innovative therapies for orphan diseases and ultra-rare pediatric disorders. Cerecor's pipeline includes several treatments for rare diseases in various stages of clinical development.

Five Biotechs Poised to Advance Their Rare Disease Programs [2022 updated]

Cruelty Free Investing

Targeting complex diseases with complex molecules

Large-molecule Therapeutics

Cerecor licenses Sanford Burnham Prebys’ Immune Checkpoint Program

Cerecor Inc., formerly known as Avalo Therapeutics, is a publicly-traded biotechnology company headquartered in Rockville, Maryland. The company focuses on developing innovative therapies for orphan diseases and ultra-rare pediatric disorders. Cerecor's pipeline includes several treatments for rare diseases in various stages of clinical development.
One of Cerecor's leading candidates is AVTX-801, an ultra-pure oral formulation of D-galactose for the treatment of phosphoglucomutase one (PGM1) deficiency, also known as PGM1-CDG. This inherited genetic disorder causes a wide range of symptoms, and AVTX-801 is entering late-stage clinical trials through an expedited 505(b)(2) pathway designation for pediatric use.
Cerecor is also targeting other rare pediatric diseases, such as MPI-CDG and Leukocyte Adhesion Deficiency Type II (LADII), with late-stage clinical trial candidates that have received expedited pathway designations. Additionally, the company has AVTX-006, a dual mTOR inhibitor in early clinical development for treating lymphatic malformations.

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

<div>
 
 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.
 
</div>

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

Cerecor

Sources

About EDGE

Contact Us

Resources

Area of Focus

Technology Research Made Effortless