Greater Philadelphia Q1 2023 News Recap – Ain’t No Stoppin’ Us Now

While times are still rocky, in Q1 Greater Philly showed that it is still very much “on the move“

By Mark Terry
April 4, 2023

A leader in cell and gene therapies, the Greater Philadelphia, or “Cellicon Valley” region, which includes Delaware and Southern New Jersey, is home to numerous top research universities and centers, such as the University of Pennsylvania, Princeton University, and The Wistar Institute.

Plenty of the biggest biopharmaceutical companies have facilities in the region, including Johnson & Johnson’s Janssen, GlaxoSmithKline and Catalent Pharma Solutions, as well as rising startups, mid-stage biotechs and CROs like Century Therapeutics, Passage Bio and the Center for Breakthrough Technologies.

And while the current economy may still be tumultuous, Greater Philly as a whole has continued to show its resilience in the first part of the new year, with several companies forging ahead into the clinic, major milestone celebrations, and construction starting for prime laboratory spaces.

Here’s a look at some of our top stories so far this year for the Philly region.

SFA Therapeutics Cleared for NASH Study

In January, Philadelphia-based SFA Therapeutics received clearance from the FDA for its IND for SFA-001N in non-alcoholic steatohepatitis (NASH) with or without fibrosis. A particularly difficult nut to crack, NASH is a form of liver inflammation and scarring (fibrosis) that can lead to cirrhosis but is not related to alcohol consumption. There are currently no FDA-approved therapies. The study will launch in the first quarter of 2023 at Einstein Medical Center in Philadelphia.

Century Therapeutics Outlined 3-Year Strategic Plan After Major Reprioritization

Speaking at the JP Morgan Healthcare conference in mid-January, Lalo Flores, PhD, CEO of Philadelphia-based Century Therapeutics, described the company’s three-year strategic plan. For this year the company will focus on advancing multiple development candidates, primarily the Phase I ELiPSE-1 clinical trial of CNTY-101 in patients with relapsed or refractory CD19+ B-cell malignancies. Earlier in the month the company reprioritized its portfolio and capital allocation to extend its cash runway. This involved deprioritizing CNTY-103 for glioblastoma and a discovery program in hematologic cancer in order to focus on CNTY-101.

Passage Bio Pushes Gene Therapy into the CNS Arena

Philadelphia-based Passage Bio has two programs currently in the clinic. The first program is PBGM01 for GM1 gangliosidosis, a genetic disorder that progressively destroys nerve cells in the brain and spinal cord. One of about 50 lysosomal storage disorders, the disease is caused by a mutation in the GLB1 gene, causing a deficiency in beta-galactosidase-1, which lysosomes require to correctly break down large sugar molecules in the body. There are currently no approved therapies to reverse the effects of the disease. The second program is PBFT02, a gene therapy for frontotemporal dementia (FTD). The most common form of dementia in people under the age of 60, FTD results in a gradual, progressive degradation in behavior, language, and movement.

Of the first trial, William Chou, M.D., CEO of Passage Bio, told BioBuzz, “We’re really pleased with our results from this trial. This is early in our program, so we’re trying to find several signals, and the first is safety. This product has a very favorable toxicity profile. There were no severe adverse events (SAEs) from the product, no immunogenicity of substance, and no AEs from our route of injection, which is through the ICM, an open area at the base of the skull.”

Liberate Bio Charges into the Nanoparticle Vector Space

The COVID-19 vaccines by Pfizer-BioNTech and Moderna showed that vaccines could be effectively delivered using lipid nanoparticles. The use of nanoparticles to deliver various payloads, such as genes, CRISPR-Cas9 therapeutics, mRNA, and nucleotides is exploding and promises to resolve many of the issues inherent with viral vectors while dramatically improving the deliverability of therapeutics.

Philadelphia-based startup Liberate Bio designs and provides nanoparticles to other biopharma companies, but acting as a provider isn’t its only focus. “The business model is actually twofold,” Nessan Bermingham, interim CEO of Liberate Bio, told BioBuzz. “We identify novel delivery vehicles — we’ll partner with other companies that need them for their respective technologies, and second, build our own pipeline where we’re utilizing them for various modalities, all nucleic acid-based, for our therapeutic areas of interest.”

Spark Broke Ground on Gene Therapy Innovation Center

In March, Spark Therapeutics broke ground on its Gene Therapy Innovation Center in University City on the Drexel University Campus in Philadelphia. Spark, now a Roche company, developed the first FDA-approved gene therapy, Luxturna. Originally announced in December 2021, Spark is investing $575 million in the innovation center located at the intersection of 30^th and Chestnut Streets. The construction will more than double Spark’s physical footprint in the Philadelphia area. The center will be 500,000 square feet and six stories. It is expected to offer increased in-house manufacturing capacity for Spark as well as for Roche and its subsidiary companies.

Palvella’s Qtorin Hits Mark in Phase II Trial

Palvella Therapeutics, headquartered in Wayne, Pa. announced positive topline data from its Phase II trial of Qtorin rapamycin in Microcystic Lymphatic Malformations, or Mycrocystic LM. The proof-of-concept study evaluated 12 people with Microcystic LMs who received the drug once a day for 12 weeks, with all showing improvement. Microcystic LM is a rare, chronically debilitating genetic disorder. It is caused by dysregulation of the PI3K/mTOR pathway and is marked by localized masses of malformed lymphatic vessels. These masses stick up through the skin and leak lymph fluid and bleed. It often causes recurrent serious infections and cellulitis. Currently, there are no FDA-approved treatments. In the U.S., about 30,000 people have been diagnosed with Microcystic LM.

Wes Kaupinen, Founder and CEO of Palvella, told BioBuzz, “If approved, QTORIN™ rapamycin has the potential to become the first therapy and standard of care for the estimated more than 30,000 individuals currently suffering from microcystic LMs in the U.S. We plan to advance to Phase 3, pending additional regulatory interactions, supported by the positive topline results of the Phase 2 study.”

BeiGene “Topped Off” New Jersey Campus and Started Hiring

BeiGene, with headquarters in Basel, Switzerland, Beijing, China and Cambridge, Mass., announced its “topping off” ceremony to celebrate the last piece of structural steel laid at its new site at Hopewell, NJ. The company has already begun hiring, expecting to bring on 100 new employees by the end of this year, with more expected by the time the construction project is completed.

The new site will be the company’s flagship U.S. biologics manufacturing and research-and-development facility. It is being constructed at the Princeton West Innovation Campus in Hopewell, NJ.

Shreya Jani, Senior Vice President of Corporate Affairs, BeiGene, told BioBuzz, “We expect the facility to be completed in the second half of 2024. The Princeton West Innovation Campus will provide state-of-the-art commercial-stage U.S. biologic pharmaceutical manufacturing, late-stage research and clinical development capabilities that complement the company’s existing capabilities around the world.”

The Innovation Space Marks 100^th Company

The Innovation Space™, headquartered in Wilmington, Del., recently marked its 100^th company when 12 new start-ups joined its Science Inc. Accelerator Program. The Innovation Space supports and “fuels” the growth of science-based startups and companies by offering a range of facilities, programs and funding opportunities. The Spring 2023 Science Inc. cohort was the largest to date and includes companies from 15 states and four countries.

Speaking with BioBuzz, The Innovation Space’s Founder, President and CEO William D. Provine, Ph.D., said, “The Science Inc. Accelerator Program allows the companies to work with a team of entrepreneurs on a journey to go from polishing their business plans to approaching investors.”