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Home » Topics » Drugs » Cell therapy

Cell therapy
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Neuronata-R

Corestemchemon preps US BLA filing for stem cell ALS therapy

June 10, 2025
By Marian (YoonJee) Chu
No Comments
Corestemchemon Inc. is planning to file a BLA for Neuronata-R (lenzumestrocel) by the end of 2025 to gain accelerated approval from the U.S. FDA, company officials confirmed to BioWorld during a June 2 interview. Neuronata-R is an autologous bone marrow-derived mesenchymal stem cell therapy that first gained approval in South Korea in 2014 to delay the progression of amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disorder also known as Lou Gehrig’s disease.
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Neuronata-R

Corestemchemon preps US BLA filing for stem cell ALS therapy

June 9, 2025
By Marian (YoonJee) Chu
No Comments
Corestemchemon Inc. is planning to file a BLA for Neuronata-R (lenzumestrocel) by the end of 2025 to gain accelerated approval from the U.S. FDA, company officials confirmed to BioWorld during a June 2 interview. Neuronata-R is an autologous bone marrow-derived mesenchymal stem cell therapy that first gained approval in South Korea in 2014 to delay the progression of amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disorder also known as Lou Gehrig’s disease.
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Regulatory, financial hurdles the gap between science and access

June 5, 2025
By Mari Serebrov
No Comments
Children with solid tumors who relapse are being treated with the same chemotherapy they would have been given 40 years ago, as “there have been no major approvals for pediatric solid tumors,” Catherine Bollard, senior vice president and chief research officer at Children’s National Hospital, said at a June 5 FDA roundtable on cell and gene therapies (CGTs). The problem isn’t the science. Bollard said many groups are working on curative CGTs “for these children who have lost all other hope for survival.” The real gap is that “big pharma doesn’t see the business model because it’s a rare disease,” she added.
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Human aging illustration

Juvenescence's $76M+ to advance CD38, ketone drugs to clinic

May 21, 2025
By Nuala Moran
No Comments
Anti-aging specialist Juvenescence Ltd. reached the first close of its series B-1 at $76 million and said it is on course to close the round at $150 million in the third quarter of 2025. “The reason for the first close and not waiting for the very end is just so we can start to move the pipeline forward,” said Richard Marshall, CEO. “We’ve got molecules in and waiting to go, so the sooner we can get going on those, the better,” he told BioWorld.
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Cancer cells being destroyed by immunotherapy
Cancer

Avidicure boosting immune activation with $50M seed round

May 19, 2025
By Nuala Moran
No Comments
Newco Avidicure NV arrived on the scene with a hefty $50 million in seed funding to advance novel antibody formats the company says will surpass the best qualities of first-generation antibodies, checkpoint inhibitors, T-cell engagers and antibody-drug conjugates.
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Illustration of kidneys with DNA double helix
Nephrology

ASGCT 2025: Overcoming kidney complexity in gene and cell therapy

May 16, 2025
By Mar de Miguel
No Comments
Gene and cell therapies (GCTs) can target the kidney to treat congenital, acute or chronic diseases affecting this organ. However, its complex structure poses a challenge for these technologies. To be precise and effective in the long term, new approaches should circumvent the specificities of renal tissue, with novel methods of delivery and gene transfer to offer new therapeutic options for patients who lack them.
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Cancer cells being destroyed by immunotherapy
Newco news

Avidicure boosting immune activation with $50M seed round

May 15, 2025
By Nuala Moran
No Comments
Newco Avidicure NV arrived on the scene with a hefty $50 million in seed funding to advance novel antibody formats the company says will surpass the best qualities of first-generation antibodies, checkpoint inhibitors, T-cell engagers and antibody-drug conjugates.
Read More
Illustration of liver with DNA double helixes
Endocrine/metabolic

ASGCT 2025: Gene and cell therapies transform metabolic diseases

May 15, 2025
By Mar de Miguel
No Comments
Metabolic disorders such as argininosuccinic and glutaric aciduria, methylmalonic acidemia, homocystinuria or primary hyperoxaluria require specific diets to prevent the accumulation of substances that the body can’t process. Current treatments mainly focus on managing symptoms and metabolite levels, and do not always prevent the progressive deterioration caused by mutations associated with the condition. However, emerging gene therapies hold promise for transforming these diseases by targeting their underlying causes, as presented in the oral abstract session, “Gene and cell therapy for metabolic diseases” of the ongoing 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) meeting in New Orleans.
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3D illustration of mesenchymal stem cells

Cynata’s MSC cells show positive results across variety of delivery systems

May 13, 2025
By Tamra Sami
No Comments
The promise of mesenchymal stem cells (MSCs) to heal heart disease could be around the corner thanks to a new delivery method tested by regenerative medicine company Cynata Therapeutics Ltd.
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3D illustration of mesenchymal stem cells

Cynata’s MSC cells show positive results across variety of delivery systems

May 9, 2025
By Tamra Sami
No Comments
The promise of mesenchymal stem cells (MSCs) to heal heart disease could be around the corner thanks to a new delivery method tested by regenerative medicine company Cynata Therapeutics Ltd.
Read More
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