Beam Therapeutics Reports Risto-Cel Shows Over 60% HbF Induction in SCD Patients
Written by Emily J. Thompson, Senior Investment Analyst
Source: Globenewswire
Updated: 1 hour ago0mins
- Significant Efficacy: In a cohort of 31 patients with severe sickle cell disease, treatment with risto-cel resulted in an average HbF level exceeding 60% and a reduction in HbS levels below 40%, highlighting the therapy's potential to improve anemia and hemoglobin levels significantly.
- Rapid Hematopoietic Reconstitution: The median time to neutrophil and platelet engraftment was 17.5 days and 19 days, respectively, with 29% of patients not requiring any platelet transfusions post-treatment, which not only reduces hospital stays but also enhances the overall patient experience.
- Efficient Cell Collection: Patients required a median of only one cell collection cycle and three total collection days for the manufacturing of risto-cel, indicating that this efficient collection and manufacturing process could lower treatment costs and improve production efficiency.
- Good Safety Profile: The safety profile of risto-cel was consistent with that of traditional autologous hematopoietic stem cell transplantation, with no severe vaso-occlusive crises reported during treatment, suggesting the therapy's feasibility and safety in clinical applications.
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Analyst Views on BEAM
Wall Street analysts forecast BEAM stock price to rise over the next 12 months. According to Wall Street analysts, the average 1-year price target for BEAM is 41.90 USD with a low forecast of 21.00 USD and a high forecast of 80.00 USD. However, analyst price targets are subjective and often lag stock prices, so investors should focus on the objective reasons behind analyst rating changes, which better reflect the company's fundamentals.
13 Analyst Rating
11 Buy
2 Hold
0 Sell
Strong Buy
Current: 28.910
Low
21.00
Averages
41.90
High
80.00
Current: 28.910
Low
21.00
Averages
41.90
High
80.00
Evercore ISI
Outperform
initiated
$35
2025-11-24
Reason
Evercore ISI
Price Target
$35
2025-11-24
initiated
Outperform
Reason
Evercore ISI initiated coverage of Beam Therapeutics with an Outperform rating and $35 price target. BEAM-302 offers "a potentially game-changing approach for AATD," with early data showing impressive levels of corrected AAT production, says the analyst, who calls upcoming data due in Q1 of 2026 "crucial."
Clear Street
Buy
maintain
$34 -> $37
2025-11-04
Reason
Clear Street
Price Target
$34 -> $37
2025-11-04
maintain
Buy
Reason
Clear Street raised the firm's price target on Beam Therapeutics to $37 from $34 and keeps a Buy rating on the shares following the Q3 report. BEAM-103 has dosed the first patient in a healthy volunteer study while the next major catalyst for the shares being the interim update from BEAM-302 in early 2026, the analyst tells investors in a research note. Clear adds that Beam owns 17% of private company Orbital Therapeutics, which amounts to $255M in its recently announced takeout.
Wedbush
David Nierengarten
Neutral
downgrade
$14 -> $9
2025-10-30
Reason
Wedbush
David Nierengarten
Price Target
$14 -> $9
2025-10-30
downgrade
Neutral
Reason
Wedbush analyst David Nierengarten lowered the firm's price target on Intellia Therapeutics (NTLA) to $9 from $14 and keeps a Neutral rating on the shares. The firm notes FDA placed a clinical hold on the Phase 3 MAGNITUDE and MAGNITUDE-2 trials of nex-z following a voluntary pause due to new safety signals of symptomatic transaminase elevation and bilirubin increase. A formal Clinical Hold Letter will be provided within 30 days. Wedbush reiterates its position that these safety signals appear target-specific rather than a class effect of LNP delivery, and views recent price action for Beam Therapeutics (BEAM) and Prime Medicine (PRME) as a buying opportunity. For Intellia's nex-z, even after the clinical hold is resolved, the firm expects reduced market uptake against competing siRNA therapies, which do not have substantial reports of hepatic toxicity and can be discontinued in the case of adverse events.
HC Wainwright & Co.
Patrick Trucchio
Strong Buy
Reiterates
$80
2025-04-07
Reason
HC Wainwright & Co.
Patrick Trucchio
Price Target
$80
2025-04-07
Reiterates
Strong Buy
Reason
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About BEAM
Beam Therapeutics Inc. is a biotechnology company developing precision genetic medicines through base editing. The Company's suite of gene editing technologies is anchored by base editing, a technology that is designed to enable precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the deoxyribonucleic acid. Its lead programs are focused on sickle cell disease and alpha-1 antitrypsin deficiency, and it is also advancing programs in other genetic diseases. Its primary programs include BEAM-101, Engineered Stem Cell Antibody Paired Evasion (ESCAPE), BEAM-302 and BEAM-301. BEAM-101 is a patient-specific, autologous hematopoietic stem cell (HSC), investigational therapy. ESCAPE is a potentially non-genotoxic approach to HSC transplantation. BEAM-302 is a liver-targeting lipid nanoparticle formulation of base editing reagents designed to offer a one-time treatment to correct the E342K point mutation (PiZZ genotype).
About the author
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Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
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