Nine CGT Therapies Poised for Regulatory Approval in 2024: Pipeline Overview from Recent Prospectus Filings

Dec 31, 2023 09:01 CST Updated 09:01

Hrain Biotechnology

Developer of Tumor Immunotherapy Technology R&D

BioCardia

Cardiovascular Disease Therapy Developer

CARsgen Therapeutics

Developer of CAR-T Cell Immunotherapy Drugs

Iovance Biotherapeutics

Developer of Novel Cancer Immunotherapies

Adaptimmune

T Cell Therapy Developer

Pfizer

Pharmaceutical R&D Developer

Autolus

T Cell Therapy Developer

Abeona Therapeutics

Cell and Gene Therapy Developer

Rocket Pharmaceuticals

Gene Therapy Developer

Disclaimer: Due to limited proficiency, errors are inevitable, and some information may not be the most up-to-date. Feel free to point out in the comments. This article only introduces drugs related to medical health and does not recommend treatment plans (if involved). This article does not constitute any investment advice.

The year 2023 is nearing its end. According to statistics,，A total of 7 cell and gene therapies received their first regulatory approvals this year, setting a new record. Looking ahead to 2024, there are 9 CGT therapies expected to gain approval.

Organized as follows:

Drug Name: Zevorcabtagene Autoleucel

R&D Company: CARsgen Therapeutics, Ltd

Zevor-cel is a fully human anti-BCMA CAR-T cell therapy product used for the treatment of relapsed/refractory multiple myeloma. In October 2022, the marketing application for Zevor-cel was accepted by the NMPA and proposed for priority review.

Company Pipeline

Drug Name:Runda Gene Therapy Injection (Proposed)

R&D Company:Shanghai Hengrun Dasheng Biotechnology Co., Ltd

December 14,CDE Official Website Announcement: Shanghai Hengrun Dasheng Biotechnology Co., Ltd.'s Runda Kymriah Injection (tentative) Marketing Application Accepted. According to the company’s earlier prospectus, the drug targets CD19, and its clinical trial application indication is for relapsed/refractory B-cell non-Hodgkin lymphoma (r/r B-NHL). Notably, the drug has already been included in the priority review process.

Shanghai Hengrun Dasheng Biotechnology Co., Ltd. Product Pipeline

Source: Hengrun Dasheng Prospectus

Drug Name:Lifileucel

R&D Company:Iovance

Lifileucel (other names: LN-144, Contego) is an off-the-shelf autologous cell therapy product containing tumor-infiltrating lymphocytes (TIL). After showing significant results in the pivotal Phase II innovaTIL-01 clinical trial, it is expected to become the first approved TIL therapy.

Company Pipeline

Drug Name:Afamitresgene autoleucel（afami-cel）

R&D Company:Adaptimmune Therapeutics

Afamitresgene autoleucel (afami-cel) is an autologous T-cell therapy where patients' T cells are modified to express a T-cell receptor (TCR) engineered to recognize melanoma-associated antigen A4 (MAGE-A4) peptides bound to MHC receptors. Patients undergo leukapheresis to collect T cells for processing and manufacturing of afamitresgene autoleucel, followed by a single infusion after lymphodepleting chemotherapy. If approved, afamitresgene autoleucel would be the first commercially available engineered T-cell therapy for solid tumor indications.

Company R&D Pipeline

Drug Name:CardiAMP

R&D Company:BioCardia

CardiAMP is the first comprehensive treatment method that utilizes a patient's own cells to treat heart failure. The treatment begins by confirming whether the patient’s bone marrow cells can be used for therapy. Subsequently, through a proprietary minimally invasive surgical procedure using a stem cell catheter, the patient's autologous bone marrow stem cells are delivered to the heart, thereby stimulating the patient's own healing mechanisms to achieve the treatment of heart failure.

Pipeline under research

Drug Name:Fidanacogene elaparvovec

Company:Pfizer

Fidanacogene elaparvovec (SPK-9001) is a novel investigational gene therapy that contains a bioengineered adeno-associated virus (AAV) capsid and a codon-optimized, highly active human factor IX gene. Once infused, it enables sustained endogenous production of factor IX in patients, thereby eliminating the need for regular factor IX infusions.

Drug Name:Marnetegragene autotemcel

R&D Company:Rocket Pharma

Gene therapy marnetegragene autotemcel (RP-L201）Autologous hematopoietic stem cells genetically modified using a lentiviral vector can enable normal expression of the ITGB2 gene. After patients receive a chemotherapy drug called busulfan, RP-L201 is reintroduced into the patient's body to eliminate existing bone marrow cells and make room for the growth of RP-L201. RP-L201 has received the FDA’s Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric Disease, and Fast Track designations, as well as PRIME and Advanced Therapy Medicinal Product (ATMP) designations in the EU, and Orphan Drug designation in both regions.

Pipeline under research

Drug Name:Obecabtagene autoleucel

R&D Company:Autolus Therapeutics

Obe-cel is Autolus' leading investigational chimeric antigen receptor (CAR) T-cell therapy for the treatment of relapsed/refractory (r/r) adult B-cell acute lymphoblastic leukemia (ALL) patients.Obe-cel has been granted the designation of "Orphan Drug" by the U.S. Food and Drug Administration (FDA), "Orphan Medicinal Product" by the European Medicines Agency (EMA), "Regenerative Medicine Advanced Therapy" (RMAT) by the U.S. Food and Drug Administration (FDA), and "Priority Medicines" (PRIME) for adult relapsed/refractory B-ALL by the European Medicines Agency (EMA).

Pipeline under research

Drug Name:Prademagene zamikeracel（pz-cel）

R&D Company:Abeona Therapeutics Inc .

Pz-cel is an investigational autologous cell therapy composed of epidermal sheets, designed to deliver the functional COL7A1 gene into the patient’s own skin cells to achieve normal expression of type VII collagen and promote wound healing. Previously, Pz-cel has been granted Regenerative Medicine Advanced Therapy (RMAT), Breakthrough Therapy, Orphan Drug, and Rare Pediatric Disease designations by the U.S. FDA.

Pipeline under research

References:

Official Websites of Various Companies