Around the Helix: Cell and Gene Therapy Company Updates – March 30, 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. Beremagene Geperpavec Improves Wound Closure in RDEB

Krystal Biotech announced positive, updated data from their phase 1/2 GEM study (NCT03536143) of beremagene geperpavec (B-VEC) that demonstrated durable wound closure in patients with recessive dystrophic epidermolysis bullosa. The therapy was well-tolerated and patients exhibited robust functional COL7 expression.

2. First Patient Dosed in Lymphoma Cell Therapy Trial

The pivotal, phase 3 E-CELERATE trial (NCT05181540) is evaluating Angiocrine Bioscience’s AB-205 cell therapy in patients with lymphoma undergoing high-dose chemotherapy and autologous hematopoietic cell transplantation. The cell therapy is intended to treat damaged organ vascular stem cell niches caused by off-target cytotoxicity and prevent progression of severe multi-organ complications.

3. Capricor’s Cell Therapy Well-Tolerated in COVID-19

The allogeneic cardiosphere-derived cell therapy CAP-1002 is being evaluated in the phase 2 INSPIRE trial (NCT04623671) in patients with COVID-19 in severe to critical condition requiring supplemental oxygen. Capricor stated that they will evaluate next steps for the program in light of declining COVID-19 hospitalizations.

4. Homology Medicines Receives Info on Clinical Hold

The company’s pheNIX trial (NCT03952156) of the gene therapy HMI-102 for the potential treatment of phenylketonuria (PKU) was placed on hold following elevated liver function test (LFT) scores in February 2022. The FDA has detailed their information request for LFTs as well as modified clinical-mitigation measures.

5. EMA Gives Positive Opinion to Cilta-Cel

The European Medicines Agency’s Committee for Medicinal Products for Human Use gave a positive opinion to Legend Biotech’s ciltacabtagene autoleucel (CARVYKTI) for relapsed/refractory multiple myeloma. The opinion was supported by positive data demonstrated in the CARTITUDE-1 trial (NCT03548207).

6. EMA Commences Review of Etrana-Dez

The EMA has commenced their review of CSL Behring's gene therapy etranacogene dezaparvovec (Etrana-Dez, AMT-061) for the potential treatment of hemophilia B. The therapy recently demonstrated efficacy in the phase 3 HOPE-B trial (NCT03569891).

7. UC Davis to Launch ADNP Syndrome Gene Therapy Program

The University of California – Davis Mind Institute is launching a program to develop therapies for activity dependent neuroprotective protein (ADNP) syndrome. The team will first develop a stem cell model of ADNP syndrome and will then evaluate potential therapeutic approaches, including adeno-associated virus vectors, antisense oligonucleotides, and CRISPR.

8. Taysha Initiates Development of Rett Syndrome Gene Therapy

Taysha’s gene therapy, TSHA-102, uses their miRARE platform to genotypically regulate transgene expression. The therapy is being evaluated in a phase 1/2 clinical trial, preliminary data from which is expected by the end of 2022. TSHA-102 is the only gene therapy in clinical development for Rett syndrome.

9. TolerogenixX to Start Phase 2b Cell Therapy Trial to Induce Immune Tolerance

The German regulatory authority Paul-Ehrlich-Institut has approved the TOL-2 clinical trial for TolerogenixX’s cell therapy MIC-Lx designed to induce immune tolerance. The trial is scheduled to start in the second quarter of 2022 and will enroll participants undergoing living donor kidney transplantation.