Around the Helix: Cell and Gene Therapy Company Updates – June 15, 2022

The cell and gene therapy sectors are growing exponentially, with new playersemerging 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.

1. bluebird Gene Therapies win Unanimous FDA Approval

The FDA's Cellular, Tissue, and Gene Therapies Advisory Committee unanimously voted in favor of the benefit-rick profiles of elivaldogene autotemcel (eli-cel; Lenti-D) for patients with early active cerebral adrenoleukodystrophy (CALD) and betibeglogene autotemcel (beti-cel; LentiGlobin) for the treatment of transfusion-dependent β-thalassemia (TDT) in adult and pediatric patients with a non–β0/β0 genotype.

2. Clinical Hold Lifted on Homology’s pheNIX Trial for Phenylketonuria

Homology Medicines has amended protocols in the pheNIX trial (NCT03952156), assessing HMI-102,to incorporate a new immunosuppression regimen with the T-cell inhibitor tacrolimus and a shorter course of steroids to address issues identified with the clinical hold placed in February 2022.

3. Freeline Doses Second Cohort in Hemophilia B Trial

Freeline Therapeutics has initiated dosing in the second cohort of their phase 1/2 B-LIEVE study (NCT05164471) of FLT180a (verbrinacogene setparvovec) for the potential treatment of hemophilia B. Updated data is expected in the second half of 2022 and first half of 2023.

4. Mustang Bio’s CAR T Therapy Shows Efficacy in Follicular Lymphoma

Mustang announced updated data from phase 1/2 clinical trial (NCT05360238) of MB-106, a CD20-targeted CAR T-cell therapy for the potential treatment of B-cell non-Hodgkin lymphomas. Data included a 94% overall response rate and 78% complete response rate in patients with follicular lymphoma.

5. Sarepta and Selecta Partnership in Muscular Dystrophies Continues

Sarepta has extended their research license and option agreement for Selecta Biosciences’ ImmTOR platform for developing therapies for Duchenne muscular dystrophy and certain limb-girdle muscular dystrophies by 9 months.

6. REGENXBIO Expands Gene Therapy Manufacturing Capabilities

The company has invested $65 million into and opened a new, state-of-the-art gene therapy manufacturing facility in Montgomery County, Maryland. The facility will boost manufacturing of the company’s NAV technology-based AAV vectors and implement its NAVXpress™ platform suspension cell culture process.

7. Charles River and ASC Therapeutics Expand Gene Therapy Agreement

The 2 companies have agreed to manufacture ASC618, a second-generation gene therapy for hemophilia A, expanding on a 3-year manufacturing agreement.

8. Center for Breakthrough Medicines Plans to Massively Expand Cell Manufacturing

The CBM, in partnership with Discovery Labs, announced that they are accelerating plans to build capacity to manufacture more than 10,000 patient therapies. The company currently provides development and manufacturing services for oncologic cell and gene therapies.

9. mCureX and ToolGen to Develop mRNA CRISPR/Cas9 Therapies for Rare Eye Diseases

mCureX, a subsidiary of OliX Pharmaceuticals, and ToolGen have signed a contract for joint research to develop gene therapies for rare eye diseases combining mCureX's mRNA technology and ToolGen's CRISPR/Cas9 gene-editing technology.

10. New Collaboration to Develop Scalable AAV Manufactuing Platform

Exothera is leading the project with LogicBio Therapeutics and Polyplus-transfection in Belgium. LogicBio will provide its mAAVRx plasmid technology and AAV experience while Polyplus brings its FectoVIR-AAV transfection reagent and process development experience to the table.

11. GenScript ProBio Licenses ProteoNic Cell Technology

ProteoNic’s 2G UNic technology is designed to boost therapeutic protein production and develop high-yield cell lines.

12. Avid Biosciences to Expand Viral Vector Manufacturing

The company has announced the launch of analytical and process development suites within its new, viral vector development, CGMP manufacturing facility. These new capabilities are expected to become available in mid-2023.