Around the Helix: Cell and Gene Therapy Company Updates – May 17, 2023

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.

1. FDA Clears Genentech and Adaptive Biotechnologies' IND for Neoantigen-Specific T-Cell Therapy

A groundbreaking collaboration between Genentech and Adaptive Biotechnologies has received FDA clearance for their investigational new drug application. The therapy shows promising potential for treating oncologic indications. The companies have been working together since 2019, leveraging Adaptive's TCR discovery platform. The CEO of Adaptive Biotechnologies expressed enthusiasm for advancing this potentially life-saving therapy into clinical trials.

2. New Hope for Dopamine Transporter Deficiency Syndrome

Bloomsbury Genetic Therapies' BGT-DTDS, an innovative gene therapy utilizing an adeno-associated virus vector, has been granted rare pediatric disease designation by the FDA. With orphan drug designation from both the FDA and European Commission, this neuron-targeted therapy aims to address the unmet needs of pediatric patients suffering from DTDS. Preclinical efficacy research has been completed, and Bloomsbury is preparing for clinical trial initiation. The company's promising development strategy, focusing on derisked capsids and accelerated clinical translation, holds potential for breakthrough treatments in multiple indications, including Parkinson disease.

3. FDA Committee Split on Accelerated Approval for Gene Therapy in Duchenne Muscular Dystrophy

Sarepta Therapeutics' gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD) has received mixed votes from the FDA Cellular, Tissue, and Gene Therapies Advisory Committee. While some committee members supported accelerated approval based on microdystrophin expression at Week 12, others expressed concerns about the study design, age subgroups, and the need for completion of confirmatory trials. Sarepta representatives addressed these concerns and emphasized the unmet needs of DMD patients. The decision marks a pivotal moment for the gene therapy and the future of DMD treatment options.

4. CytoAgents' CTO1681 Receives FDA Clearance for Investigational New Drug Application

CytoAgents has gained FDA clearance for its small molecule drug CTO1681, aimed at managing cytokine release syndrome (CRS). The drug targets the NF-kB signaling pathway to reduce inflammation and modulate cytokine production. With potential applications in CRS caused by CAR-T therapy, bispecific antibody therapy, COVID-19, and respiratory diseases, CytoAgents plans to initiate a phase 1b/2a clinical trial for CRS management in CAR-T therapy patients this summer. The clearance marks a significant milestone for the company's mission to prevent and treat CRS, a life-threatening adverse effect of immunotherapies.

5. uniQure Sells Portion of Hemgenix Royalty Rights

uniQure has reached an agreement to sell a portion of its royalty rights from CSL Behring's sales of Hemgenix gene therapy. The deal, valued at up to $400 million, involves upfront cash payments of $375 million from HealthCare Royalty and Sagard Healthcare. uniQure will receive royalties on CSL's global sales of Hemgenix until 2038, with additional payments if 2024 net sales exceed a specified level. The transaction will provide immediate capital for uniQure's gene therapy pipeline and advancement of breakthrough therapies for conditions like Huntington disease, epilepsy, and amyotrophic lateral sclerosis.

6. Ray Therapeutics Raises $100 Million for Vision-Restoring Gene Therapies

Ray Therapeutics has secured $100 million in Series A financing to advance its gene therapies aimed at restoring visual function in individuals with blinding diseases. The biotechnology startup plans to use visual optogenetics to deliver light-sensitive proteins into the eye, converting retinal neurons into photoreceptors. By doing so, Ray aims to compensate for the loss of photoreceptors and restore visual signals to the brain. The funding will support the clinical testing of its lead program for retinitis pigmentosa, with plans to expand into other retinal degenerative diseases. The approach has the potential to treat various genetic mutations associated with blindness.

7. Eli Lilly's Prevail Therapeutics Unit Secures Rights to CRISPR X-Editing Technologies

Eli Lilly's Prevail Therapeutics has entered into an agreement with Scribe Therapeutics to develop genetic medicines for neurological and neuromuscular diseases using Scribe's CRISPR X-Editing technologies. Scribe's platform, based on its CRISPR by Design approach, offers in vivo genome editing capabilities with enhanced safety, efficacy, and delivery. The deal includes an upfront payment, equity investment, and milestone payments totaling over $1.5 billion, along with R&D funding and tiered royalties. Scribe also has a partnership with Sanofi for genetic modification of novel natural killer cell therapies for cancer.

8. Gene Therapies for Rare Diseases Accelerated by National Consortium

The Foundation for the National Institutes of Health (FNIH) has partnered with drugmakers and nonprofits to accelerate the development of gene therapies for rare diseases. The Bespoke Gene Therapy Consortium has selected eight rare diseases, including ocular, neurological, and systemic conditions, for its research focus. The consortium aims to streamline the path to clinical trials for therapies that use adeno-associated viral vectors (AAVs). With over $100 million in financial and in-kind commitments from more than 30 organizations, including Biogen, Johnson & Johnson, and Pfizer, the consortium seeks to create standardized solutions and a publicly available roadmap for AAV gene therapies.

9. Gilead Names New Executive Vice President of Kite

Gilead Sciences has announced the appointment of Cindy Perettie, a former Roche executive, as the new head of its cell therapy business, Kite Pharma. Perettie brings more than 20 years of experience in scientific and commercial leadership in oncology to her new role, and will be responsible for driving the growth of Kite Pharma, which produces cancer cell therapies such as Yescarta and Tecartus. Her appointment comes after Christi Shaw stepped down from the position in March 2023. Perettie's expertise will be crucial in navigating the competitive landscape of cancer cell therapy.

10. Sangamo Loses Collaborators on Neurological, Neurodevelopmental Gene Therapy Programs

Sangamo Therapeutics has experienced setbacks as two of its partnerships, first with Novartis and then with Biogen, have been terminated. The collaborations aimed to explore potential gene targets for neurological and neurodevelopmental disorders using Sangamo's zinc finger protein transcription factors (ZFP-TFs). Following the terminations, Sangamo will review its preclinical programs and seek a partner to continue development. The company recently redirected its resources to other clinical-stage programs, including gene therapy for Fabry disease and an autologous CAR-Treg therapy. Sangamo remains committed to advancing its pipeline and delivering innovative treatments for patients in need.

11. MSLN-Targeted KIR-CAR Therapy Trial Initiates at UPenn

Verismo Therapeutics has initiated the phase 1 STAR-101 clinical trial to evaluate SynKIR-110 in patients with mesothelin (MSLN)-expressing pleural mesothelioma, cholangiocarcinoma, or ovarian cancer. SynKIR-110, developed using Verismo's KIR-CAR platform, combines an MSLN-specific antibody with natural killer cell signals to target MSLN-expressing tumors. The trial aims to assess safety, feasibility, preliminary efficacy, biomarkers, and a recommended phase 2 dose of SynKIR-110. The therapy has received fast track designation from the FDA, marking an important milestone in bringing this potentially life-saving treatment to patients in need. Verismo Therapeutics is committed to finding new treatments for these devastating diseases and providing hope for improved outcomes.