Weekly Quick Hits (Greater Philly)
– Week of February 27, 2023

By Mark Terry, Alex Keown, and Sarah Ellinwood
March 3rd, 2023

The Philadelphia life science market is as hot as ever, and “Cellicon Valley” is generating plenty of news. Here’s a look at this week’s stop stories…

Funding and Collaborations

Spark Therapeutics Begins Construction of Gene Therapy Innovation Center

Spark Therapeutics, a Roche company based in Philadelphia, initiated construction of its $575 million Gene Therapy Innovation Center. The site is on the Drexel University campus in Philadelphia’s University City neighborhood. The new location is projected to be 500,000 square feet.

Arbutus Reports Q4 and 2022 Financials

Warminster, Pa.-based Arbutus Biopharma reported its 2022 and Q4 financials, emphasizing three key initiatives: exploring combination therapies with AB-729, its RNAi therapeutic for hepatitis B virus; advancing its preclinical HBV compounds AB-101, its oral LPD-L1 inhibitor, and AB-161, its oral RNA destabilizer; and identifying a clinical candidate that inhibits the SARS-CoV-2 nsp5 main protease. Total revenue was $39 million for the year and the company has $184.3 million in cash as of December 31, 2022.

In the Clinic

CSL’s Phase III Trial of Garadacimab for HAE Hits Mark

CSL, based in King of Prussia, Pa., reported that its Phase III VANGUARD study of garadacimab for hereditary angioedema (HAE) significantly reduced the attack rate compared to placebo. Garadacimab is a first-in-class monoclonal antibody developed as a long-term prophylactic treatment for HAE.

New Products

CBM Announces Pre-Clinical Vector Manufacturing Package

The Center for Breakthrough Medicines, a CDMO based in King of Prussia, Pa., announced it is packaging their pre-clinical capabilities into an IND-enabling offering. This package is capable of delivering end-to-end support for preclinical studies and includes complimentary off-the-shelf AAV rep-cap and helper plasmids, custom plasmid manufacturing, access to a proprietary cell line and more.

Research Roundup

Princeton Research: A Key Protein in Hepatitis E

Researchers from Princeton’s Department of Molecular Biology published a study in eLife describing a new model of the structure and function of a Hepatitis E protein. The RNA virus results in about 70,000 deaths per year and 3 million symptomatic infections. Hep E is usually transmitted via fecal contamination of water, food or surfaces.

Palatin Published Preclinical Data About its Inflammatory Bowel Disease Therapy

Palatin Technologies, headquartered in Cranbury, NJ, published a preclinical study of its melanocortin-1 receptor agonist PL8177 in the journal Frontiers in Immunology. The drug is being developed for inflammatory bowel disease.

Jefferson Research: New Target in Triple-Negative Breast CancerA research team at Jefferson Health’s Sidney Kimmel Cancer Center developed a novel therapeutic strategy for triple-negative breast cancer targeting p53-inactivated TNBC. They found that deletion of the Mdm2 gene induced death of mouse cancer cells lacking p53.

Did we miss something?

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