FDA grants RMAT designation to Beam Therapeutics’ sickle cell therapy

CAMBRIDGE, Mass. - The U.S. Food and Drug Administration has granted Regenerative Medicine Advanced Therapy (RMAT) designation to BEAM-101, Beam Therapeutics Inc.’s (NASDAQ:BEAM) investigational cell therapy for sickle cell disease, the company announced Thursday. According to InvestingPro data, Beam Therapeutics, currently valued at $1.72 billion, appears slightly undervalued based on its Fair Value analysis.

The designation follows orphan drug status awarded in June and provides opportunities for accelerated development pathways, including early FDA interactions, potential surrogate endpoints for accelerated approval, and possible priority review. While the stock has declined nearly 30% year-to-date, analysts maintain a bullish outlook with price targets ranging from $20 to $80 per share. Get deeper insights into Beam’s potential with InvestingPro’s comprehensive research report, one of 1,400+ available for top US stocks.

BEAM-101 is a genetically modified cell therapy that uses base editing to increase production of fetal hemoglobin, which can reduce sickling of red blood cells. The treatment involves collecting a patient’s own blood stem cells, editing them, and returning them via a stem cell transplant procedure.

Updated data from the ongoing BEACON Phase 1/2 trial presented at the European Hematology Association Congress in June showed that 17 treated patients experienced increases in fetal hemoglobin, reductions in sickle hemoglobin, and normalized markers of hemolysis and oxygen delivery. No vaso-occlusive crises were reported after engraftment, according to the company’s press release statement.

"These designations not only recognize the promise of BEAM-101 but also enable enhanced collaboration with the FDA as we advance toward a BLA filing," said Giuseppe Ciaramella, president of Beam Therapeutics. The company maintains a strong financial position with a current ratio of 6.75, though InvestingPro analysis indicates its overall financial health score as ’FAIR’.

The company reported that 30 patients have now been dosed in the trial, with additional data expected later this year.

Sickle cell disease affects approximately 100,000 people in the United States and 8 million worldwide. The genetic blood disorder causes red blood cells to form a sickle shape, leading to blocked blood vessels, severe pain, organ damage, and shortened lifespan.

In other recent news, Beam Therapeutics reported its second-quarter 2025 financial results, with earnings of -$1.00 per share, surpassing the consensus estimate of -$1.11 per share. Clear Street has maintained its Buy rating for Beam Therapeutics, setting a price target of $34. Additionally, H.C. Wainwright reiterated its Buy rating with a higher price target of $80, highlighting the company’s innovative base editing pipeline. Beam’s BEAM-101 therapy for sickle cell disease (SCD) has shown promising results in the BEACON Phase 1/2 trial, achieving significant increases in fetal hemoglobin and reductions in sickle hemoglobin. The therapy’s performance is considered a benchmark for therapeutic success in treating SCD. Furthermore, the FDA has granted orphan drug designation to BEAM-101, facilitating its development for rare diseases. This designation supports the ongoing clinical trials, which have demonstrated a positive safety profile. Beam Therapeutics continues to generate interest from investors, as noted by H.C. Wainwright during virtual investor meetings.

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