Around the Helix: Cell and Gene Therapy Company Updates – August 27, 2025

The cell and gene therapy sectors are growing exponentially, with new players emerging daily and much progress being made both in and out of the lab. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.

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1. FDA Approves Precigen’s Recurrent Respiratory Papillomatosis Gene Therapy Papzimeos

The FDA has approved Precigen’s zopapogene imadenovec-drba (also known as PRGN-2012), a nonreplicating adenoviral vector-based immunotherapy, for the treatment of adults with recurrent respiratory papillomatosis (RRP).

2. FDA Lifts Clinical Hold on Rocket’s Trial for Danon Disease Gene Therapy RP-A501

The FDA has lifted its clinical hold on Rocket Pharmaceuticals’ pivotal phase 2 clinical trial (NCT06092034) evaluating RP-A501, an investigational adeno-associated virus serotype 9 (AAV9) vector-based gene therapy intended to treat Danon disease.

3. Cilta-Cel's Efficacy in Second-Line Relapsed/Refractory Multiple Myeloma

Surbhi Sidana, MD, an associate professor of medicine and associate director for Clinical Research in the Bone Marrow Transplantation and Cell Therapy Division at Stanford University, spoke to CGTLive®'s sister site OncLive® about the efficacy results of the phase 3 CARTITUDE-4 clinical trial (NCT04181827).

4. Arnatar Therapeutics' ART4 Snags FDA RPDD and Orphan Drug Designation

Arnatar, an RNA therapy-focused company that recently emerged from stealth, announced along with its emergence that it has received rare pediatric disease designation (RPDD) and orphan drug designation from the FDA for ART4, an investigational up-regulating antisense oligonucleotide (ASO) therapy intended to treat Alagille syndrome (ALGS). “Our approach is designed to target the underlying genetic deficiency to restore bile duct function and prevent the severe complications, including liver damage, that children with ALGS often face," Xuehai Liang, the chief executive officer of Arnatar, said in a statement.1 "We remain committed to advancing ART4 into the clinic as rapidly as possible to bring this potentially transformative therapy to patients and families who currently have no curative treatment options.”

5. Prerna Mewawalla, MD, on Unmet Needs Following Early-Line CAR-T for R/R Multiple Myeloma

The medical director of Apheresis and a hematologist-oncologist in the Division of Hematology and Cellular Therapy at Allegheny Health Network, as well as an associate professor at the Drexel University College of Medicine, spoke about unmet needs and emergent questions that come up after CAR-T therapy is used to treat patients for r/r MM in earlier line settings.

6. Appia Bio Goes Under

According to a LinkedIn post from chief executive officer JeenJoo S Kang, PhD, Appia Bio, a company focused on the development of allogeneic and "broadly accessible" chimeric antigen receptor T-cell (CAR-T) therapy, has shut down its operations. Kang cited a lack of funding for entering clinical stage development as the reason for the company shuttering its doors, noting that it had been nearing filing of an investigational new drug application.

7. Sarepta Refinancing After Recent Struggles

Following a series of challenges regarding its Duchenne muscular dystrophy (DMD) gene therapy delandistrogene moxeparvovec-rokl (marketed as Elevidys), which was briefly pulled from the market this summer before later resuming shipping, Sarepta Therapeutics has announced a refinancing of around $700 million of its 1.25% convertible senior notes that are due in 2027. "By extending the maturity of a meaningful portion of our convertible notes to 2030, we have completed a shareholder-friendly transaction that significantly enhances our balance sheet flexibility and strengthens our financial position," Doug Ingram, JD, the president and chief executive officer of Sarepta, said in the statement. "With our go-forward cash flows and liquidity, we believe we are well positioned to fully fund our pipeline and meet our near-term obligations. This allows us to focus on our base business and the upcoming readouts from our pipeline, including our siRNA programs, as we continue to pursue our mission to transform the lives of patients with rare diseases."