Eight Philadelphia biotech companies spearheading the industry – Labiotech.eu

Philadelphia has rapidly emerged as a major hub for biotechnology, often referred to as Cellicon Valley due to its significant contributions to cell and gene therapy. This thriving ecosystem is driven by a combination of leading academic institutions, a robust network of research organizations, and a dynamic startup scene. Key institutions like the University of Pennsylvania and Childrens Hospital of Philadelphia have been instrumental in developing groundbreaking therapies, such as the first Food and Drug Administration (FDA)-approved gene therapy, Luxturna.Here are eight biotech companies based in Philadelphia to put on your radar.

AUM LifeTech, founded in 2013, is a Philadelphia-based preclinical biotech company. It focuses on developing RNA silencing technologies. Their proprietary FANA technology offers a next-generation approach to gene silencing and regulation. FANA antisense oligonucleotides (ASOs) are designed to target and silence specific RNA molecules, including mRNA, microRNA, and long non-coding RNA, without requiring delivery agents such as viral vectors or complex formulations. ASOs are short, synthetic strands of nucleic acids designed to specifically bind to the mRNA of a target gene. By binding to this mRNA, ASOs can modify the expression of the target gene. This self-delivering capability enhances the efficiency and reduces the toxicity of RNA silencing applications, making it particularly useful in therapeutic development and biomedical research.

AUM LifeTech has established key collaborations with institutions such as the Childrens Hospital of Philadelphia and the University of Pennsylvania. These partnerships aim to leverage FANA technology in cancer immunotherapy and other therapeutic areas.

AUM LifeTechs pipeline focuses on developing RNA silencing therapies for a variety of genetically defined diseases. The company also has a T-regulatory (Treg) candidate focused on lung cancer targeting the FOXP3 gene. AUM LifeTech also develops a project targeting Parkinsons disease.

BlueWhale Bio, a biotech company founded in 2023 and based in Philadelphia, emerged from the University of Pennsylvania. It focuses on cell and gene therapy manufacturing. BlueWhale Bio aims to overcome the bottlenecks in the production of cell and gene therapies to make these treatments more accessible and affordable for patients.

In September 2023, BlueWhale Bio secured $18 million in seed financing led by Danaher Ventures. This initial funding supports the development of BlueWhale Bios manufacturing platform and product portfolio.

While specific product details about the companys pipeline are still under wraps, the companys approach focuses on addressing the critical needs of cell and gene therapy production, including improving process efficiency, scalability, and cost-effectiveness.

The company is the first to emerge from the Danaher Ventures Pioneer Program, which supports the creation of companies with disruptive technologies in life sciences.

This is not the first time we have talked about Carisma Therapeutics as it was on our list of companies to follow last December. This biotech company based in Philadelphia is innovating in the field of immunotherapy with its development of chimeric antigen receptor macrophages (CAR-M) and CAR-monocytes. These engineered cells leverage the innate and adaptive immune responses to target and destroy cancer cells, particularly in solid tumors. Unlike traditional CAR-T therapies that face challenges in treating solid tumors, CAR-macrophages are designed to overcome these hurdles by infiltrating tumors, surviving in the hostile tumor environment, and activating a broader immune response.

Carisma raised substantial capital through multiple funding rounds, bringing the total to $151.9 million after completing a $30 million series C round in 2002.

Additionally, Carisma has established a collaboration with Merck to evaluate the efficacy of CAR-macrophages in combination with Mercks anti-PD-1 therapy, KEYTRUDA. This collaboration aims to explore the synergistic effects of combining CAR-M technology with existing immunotherapies to enhance treatment outcomes for HER2-positive cancers. The companies announced the dosing of the first patient in a phase 1 clinical trial in 2023.

In 2024, Carisma made strategic decisions to streamline its pipeline. While development of its CAR-Macrophage candidate CT-0508 has been halted, the company continues to advance CT-0525, a CAR-Monocyte that received FDA clearance in 2023. This clearance allows Carisma to begin evaluating CT-0525 in phase 1 clinical trials. Indeed, the company announced the dosing of the first patient in phase 1 clinical study, last week.

Century Therapeutics, founded in 2018 and based in Philadelphia, is a clinical-stage biotech company that leverages induced pluripotent stem cells (iPSCs) to develop allogeneic, or off-the-shelf, cell therapies. IPSCs are a type of stem cell generated directly from adult cells. They are created by reprogramming somatic cells, such as skin or blood cells, back into an embryonic-like pluripotent state. They offer a renewable source of various cell types, which can be used for therapeutic purposes, drug testing, and understanding disease mechanisms without the ethical concerns associated with embryonic stem cells.

These therapies aim to treat cancers and autoimmune and inflammatory diseases. Centurys approach utilizes the self-renewing capacity of iPSCs combined with advanced genetic engineering and manufacturing technologies.

In April 2024, the company closed a $60 million private placement being utilized to expand the clinical development of their lead program, CNTY-101, among other initiatives. Additionally, Century Therapeutics has a collaboration with Bristol-Myers Squibb.

CNTY-101, the companys lead program is an iPSC-derived NK (natural killer) cell therapy targeting CD19-positive hematologic cancers. It is currently in clinical development and has shown promising preliminary phase 1 results in inducing cytolysis of B-cells. The company plans to expand CNTY-101s application into additional autoimmune disease indications.

Century is also advancing other iPSC-derived NK and T cell therapies targeting various cancers and autoimmune diseases, leveraging their platform to develop multiple product candidates with the potential to provide significant therapeutic benefits.

In 2024, Century Therapeutics announced the acquisition of Clade Therapeutics, which enhances its capabilities in developing scalable, consistent stem cell-based medicines.

Context Therapeutics, headquartered in Philadelphia, is a clinical-stage biotech company focused on developing treatments for solid tumors. The companys primary focus is on hormone-driven cancers, including breast, ovarian, and endometrial cancers. Contexts lead product candidate, CTIM-76, is a selective Claudin 6 (CLDN6) x CD3 bispecific antibody designed to target CLDN6-positive tumors. CLDN6 is a membrane protein found in multiple solid tumors but is minimally expressed in healthy adult tissues, making it an attractive target for cancer therapy.

In May 2024, Context Therapeutics announced the completion of a $100 million private placement. This funding is expected to support the companys operations into 2028, providing substantial resources for the development and clinical testing of CTIM-76 and other pipeline candidates. The company has also received FDA clearance for its IND application for CTIM-76, allowing it to proceed with a phase 1 clinical trial.

iECURE, founded in 2020 and based just outside of Philadelphia, is a clinical-stage gene editing company dedicated to developing mutation-agnostic in vivo gene insertion therapies for treating liver disorders. These therapies aim to knock in healthy copies of disease-causing genes, meaning, introducing a functional copy of a gene directly into the genome of a patients cells. The companys approach targets a wide range of genetic mutations, making it a versatile solution for many monogenic liver diseases.

iECURE has raised significant funding with a notable $50 million series A round, followed by an additional $65 million in series A-1 financing. The company has also received FDA Fast Track designation and orphan drug designation for its lead product candidate, ECUR-506, allowing the initiation of the OTC-HOPE clinical trial. Additionally, the company received approval from the U.K. Medicines & Healthcare Products Regulatory Agency (MHRA) to expand this study.

ECUR-506, the companys lead candidate targets ornithine transcarbamylase (OTC) deficiency, a severe inherited metabolic disorder. ECUR-506 utilizes two adeno-associated virus (AAV) capsids: one carrying an ARCUS nuclease to target gene editing in the PCSK9 gene, and another carrying the functional OTC gene. This innovative approach aims to provide permanent expression of the healthy OTC gene. The OTC-HOPE study, a phase 1/2 clinical trial, is currently enrolling newborn males with severe neonatal onset OTC deficiency to evaluate the safety, tolerability, and efficacy of ECUR-506.

Passage Bio, founded in 2019 is developing transformative therapies for patients suffering from central nervous system (CNS) disorders. The companys primary focus is on creating one-time gene therapies designed to address the underlying pathology of neurodegenerative diseases. Passage Bios lead product candidate, PBFT02, aims to treat frontotemporal dementia (FTD) in patients with mutations in the GRN gene, by elevating progranulin levels to restore lysosomal function and slow disease progression.

PBFT02 uses a harmless virus (AAV1) to carry a healthy copy of the GRN gene directly to the brain. The virus is injected into a fluid-filled space at the base of the brain, the cisterna magna. This method aims to deliver the gene precisely where its needed to help restore normal brain function and slow down the diseases progression.

In 2024, Passage Bio reported positive interim results from the upliFT-D clinical trial for PBFT02. The data showed that the therapy was generally well-tolerated with no serious adverse events in patients receiving an enhanced steroid regimen. Additionally, significant biomarker improvements were observed. Passage Bio plans to expand the clinical development of PBFT02 into additional neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS) and Alzheimers disease.

Passage Bio is also developing gene therapies for GM1 gangliosidosis, Krabbe disease, metachromatic leukodystrophy (MLD), and other neurodegenerative conditions.

Verismo Therapeutics, founded in 2022 and based in Philadelphia, is a clinical-stage biopharmaceutical company working on the development of next-generation CAR T-cell therapies using its novel KIR-CAR platform. This platform incorporates killer immunoglobulin-like receptors (KIRs) derived from NK cells, aiming to improve T-cell persistence and efficacy, especially in challenging solid tumor environments.

KIRs are a family of receptors found on the surface of NK cells, which are part of the innate immune system. These receptors help NK cells recognize and respond to infected or cancerous cells by binding to specific ligands on the target cells. KIRs regulate the activity of NK cells by either activating or inhibiting their functions. When KIRs bind to their specific ligands, they can enhance the cytotoxic activity of NK cells against tumor cells, making them a critical component in targeting cancer cells.

Verismos approach seeks to address the limitations of traditional single-chain CAR T-cell therapies, such as T-cell exhaustion and limited efficacy in solid tumors.

Verismo Therapeutics has secured notable funding, including a $17 million pre-series A financing round. The company collaborates closely with the University of Pennsylvania, where the KIR-CAR technology was originally developed. This partnership includes access to research and clinical trial support.

Verismos pipeline features several promising candidates:

The citys strategic location along the U.S. Northeast corridor offers easy access to major markets, investors, and collaborators, making it an attractive base for biotech companies. Philadelphias biotech landscape is characterized by its collaborative environment where academic institutions, research centers, and biotech companies work closely together. This synergy is evident in initiatives like the Keystone LifeSci Collaborative, which focuses on building a coordinated strategy for industry growth, talent development, and regional competitiveness.

Philadelphias biotech sector is supported by substantial investments and state-of-the-art infrastructure. The University City Science Center and the Navy Yard, home to 150 companies, are examples of dedicated biotech hubs within the city, providing space and resources for innovation and development.

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Eight Philadelphia biotech companies spearheading the industry - Labiotech.eu