Victoria Johnson joined the MJH Life Sciences in 2020 and has written for NeurologyLive, CGTLive, and now HCPLive. You can reach her at vjohnson@mjhlifesciences.com

Topline data from the phase 3 PROSERA trial indicate that seralutinib, an investigational inhaled tyrosine kinase inhibitor, did not meet its prespecified primary efficacy threshold in 

(PAH), despite achieving nominal statistical significance on 6-minute walk distance (6MWD) at 24 weeks and higher efficacy in patients with intermediate and high-risk PAH.¹ 

“While we are disappointed to have narrowly missed the stringent prespecified statistical threshold for our primary endpoint, the result still clears the traditional 0.05 p-value, and we believe these data clearly demonstrate seralutinib is an active drug in patients with PAH,” Faheem Hasnain, Chairman, Co-Founder, and CEO of Gossamer Bio, said in a statement.

According to Gossamer Bio, the randomized, double-blind, placebo-controlled PROSERA study enrolled 390 patients with World Health Organization (WHO) functional class II or III PAH receiving background therapy.¹ Patients were randomized to seralutinib (n = 197) or placebo (n = 193) for up to 48 weeks. At week 24, the placebo-adjusted Hodges-Lehmann median difference in 6MWD was +13.3 meters (

= .0320), which did not meet the prespecified alpha threshold of 0.025 for the primary endpoint.¹ Because the primary endpoint was not met at the adjusted alpha level, secondary endpoint p values are considered nominal.

Median change from baseline in 6MWD was +28.2 meters in the seralutinib group and +13.5 meters in the placebo group at week 24.¹ In prespecified patients with intermediate- and high-risk PAH (REVEAL Lite 2 score ≥6; n = 234), the placebo-adjusted improvement was +20.0 meters (

 = .0207), an efficacy signal that was consistent with data seen from the phase 2 TORREY trial.

 Changes in N-terminal pro–B-type natriuretic peptide (NT-proBNP) at week 24 favored seralutinib (estimated location shift −120.4 ng/L; 

 = .0002), and other secondary measures—including clinical improvement and ≥1-point REVEAL Lite 2 score reduction—also numerically favored active treatment.¹ Time-to-clinical worsening hazard ratio was 0.744 (

“We are also pleased by the clinically meaningful improvements observed in intermediate- and high-risk patients who are at an increased risk of significant morbidity and mortality events and represent a population with a high unmet need. From a clinical development perspective, this is not a narrow or exploratory finding. Seralutinib has once again demonstrated a statistically robust and clinically meaningful signal in higher‑risk patients, consistent with the TORREY Study, which is a clearly defined and readily identifiable population. This finding is compelling on its own,” Hasnain said.

Seralutinib is an inhaled inhibitor of platelet-derived growth factor receptor (PDGFR), colony-stimulating factor 1 receptor (CSF1R), and c-KIT, pathways implicated in vascular proliferation and remodeling.

 Unlike established PAH therapies, which primarily target vasodilation, seralutinib is designed to address proliferative and inflammatory mechanisms. Phase 2 data from the TORREY study previously demonstrated improvements in pulmonary vascular resistance and exploratory clinical endpoints which supported advancement to phase 3 evaluation.

 Inhaled tyrosine kinase inhibitor (PDGFR, CSF1R, c-KIT)

 PROSERA, phase 3, randomized, placebo-controlled (n = 390)¹

 Placebo-adjusted 6MWD +13.3 m at Week 24 (

 Transaminase elevations ≥3× ULN in 13% vs 1% with placebo¹

 Investigational; sponsor plans FDA discussion¹

Seralutinib was generally well tolerated. Treatment-emergent adverse events (TEAEs) occurred in 86.5% of patients receiving seralutinib and 80.5% receiving placebo.¹ Serious adverse events occurred in 16.0% and 18.9%, respectively. Transaminase elevations ≥3 times the upper limit of normal were reported in 13% of seralutinib-treated patients versus 1% with placebo.¹ Cough was the most frequent AE (37.0%).

PAH remains a progressive and life-threatening condition characterized by pulmonary vascular remodeling and right ventricular failure.

 Contemporary treatment algorithms emphasize risk stratification and early combination therapy with endothelin receptor antagonists, phosphodiesterase-5 inhibitors or soluble guanylate cyclase stimulators, and prostacyclin pathway agents. Despite therapeutic advances, morbidity and mortality remain substantial, particularly among intermediate- and high-risk patients. Hasnain stressed that the PROSERA population reflects the difficult-to-treat reality in contemporary practice: 55% were receiving triple or quadruple background therapy and 61% were on prostacyclin treatment.¹ 

Although the primary endpoint narrowly missed the prespecified statistical threshold, the nominally positive 6MWD result and consistent signal in higher-risk subgroups suggest biological activity. Gossamer Bio has indicated plans to meet with the United States Food and Drug Administration (FDA) to discuss a potential path forward.¹ 

Gossamer Bio. Gossamer Bio announces topline results from the Phase 3 PROSERA study evaluating seralutinib in pulmonary arterial hypertension. February 23, 2026. Accessed February 23, 2026. https://www.gossamerbio.com/news-releases/news-release-details/gossamer-bio-announces-topline-results-phase-3-prosera-study

Humbert M, Kovacs G, Hoeper MM, et al. 2022 ESC/ERS Guidelines for the diagnosis and treatment of pulmonary hypertension. 

. 2022;43(38). doi: 10.1093/eurheartj/ehac237

Frantz RP, McLaughlin VV, Sahay S, et al. Seralutinib in adults with pulmonary arterial hypertension (TORREY): a randomised, double-blind, placebo-controlled phase 2 trial. 

. 2024;12(7):523-534. doi: 10.1016/S2213-2600(24)00072-9

Articles

Phase 3 PROSERA evaluated inhaled seralutinib added to background therapy in WHO FC II–III PAH. The study narrowly missed its prespecified alpha (0.025) for the 24-week 6MWD primary endpoint despite nominal significance (+13.3 m; P=.032). A stronger, consistent signal emerged in intermediate/high-risk patients (REVEAL Lite 2 ≥6; +20.0 m; P=.0207) alongside favorable NT-proBNP shifts. Safety was generally acceptable, but transaminase elevations were more frequent. Regulatory discussions are planned.

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PROSERA: Seralutinib Falls Short in Pulmonary Arterial Hypertension Phase 3 Trial

Alex Hillenbrand is an assistant editor for 

. She earned her BA in English from Lafayette College in 2023 and went on to write for pop culture, entertainment, and sports media outlets before joining MJH Life Sciences in 2025. In her free time, Alex enjoys cooking, going on walks, and meeting new people. Contact her at mjhlifesciences.com.

Luspatercept (Reblozyl®) met primary endpoints in both non-transfusion-dependent (NTD) and transfusion-dependent (TD) adults with 

 in the first registrational phase 2 trial in this population.

Bristol Myers Squibb announced the positive top-line results on February 23, 2026, including luspatercept increased hemoglobin in NTD and reduced transfusion burden in TD, along with meeting all secondary endpoints and exhibiting a consistent safety profile.

“These positive data further support the potential of Reblozyl for patients around the world,” said Cristian Massacesi, MD, Executive Vice President, Chief Medical Officer, and Head of Development at Bristol Myers Squibb, in a statement. “This is the first and only registrational Phase 2 trial specifically designed to address the needs of patients, especially in China, with alpha-thalassemia, a lifelong disease with limited treatment options and the potential for serious long-term complications.”

Alpha-thalassemia is a hereditary blood disorder characterized by reduced or absent alpha-globin chain production, leading to chronic anemia. Patients with transfusion-dependent disease often require lifelong RBC transfusions, which are associated with iron overload and other complications. Therapeutic options for non–transfusion-dependent alpha-thalassemia remain limited.

Luspatercept is a recombinant fusion protein that binds select transforming growth factor-β superfamily ligands, inhibiting SMAD2/3 signaling and promoting late-stage RBC maturation. In the United States, Reblozyl is approved by the US Food and Drug Administration for anemia in adults with beta-thalassemia who require regular RBC transfusions and for certain lower-risk myelodysplastic syndromes. It is not currently approved for alpha-thalassemia.

In the NTD cohort, luspatercept demonstrated a statistically significant and clinically meaningful increase in hemoglobin. The primary endpoint was a ≥1 g/dL increase from baseline in mean hemoglobin over a continuous 12-week interval from Week 13 to Week 24 in the absence of red blood cell (RBC) transfusion.

In the TD cohort, luspatercept demonstrated a statistically significant and clinically meaningful reduction in RBC transfusion burden. The primary endpoint was a ≥50% reduction from baseline in transfusion burden with a ≥2-unit reduction during any continuous 12 weeks between Week 13 and Week 48 compared with the 12-week interval before the first dose.

The ongoing study is evaluating luspatercept plus best supportive care versus placebo in adults and adolescents with alpha-thalassemia, including both TD and NTD cohorts. The adolescent cohorts remain ongoing. Data from the trial are expected to be presented at an upcoming medical congress and will be discussed with the Center for Drug Evaluation in China.

Bristol Myers Squibb Announces Positive Top-Line Results from Registrational Phase 2 Study of Luspatercept in Adults with Alpha (α)-Thalassemia. Bms.com. Published 2020. Accessed February 23, 2026. 

https://news.bms.com/news/details/2026/Bristol-Myers-Squibb-Announces-Positive-Top-Line-Results-from-Registrational-Phase-2-Study-of-Luspatercept-in-Adults-with-Alpha--Thalassemia/default.aspx

Patel B, Moosavi L. Luspatercept. PubMed. Published 2023. 

https://www.ncbi.nlm.nih.gov/books/NBK560635/

Positive top-line results from a registrational phase 2 trial showed luspatercept met primary endpoints in adults with alpha-thalassemia across non–transfusion-dependent and transfusion-dependent cohorts. Non–transfusion-dependent patients achieved a statistically significant, clinically meaningful hemoglobin rise without interim RBC transfusions, while transfusion-dependent patients had substantial reductions in transfusion burden. All secondary endpoints were met with a safety profile consistent with prior experience. The trial includes best supportive care and placebo comparators, with adolescent cohorts ongoing and planned regulatory discussions in China.

Luspatercept met primary endpoints in transfusion-dependent and non–transfusion-dependent alpha-thalassemia.

Hematology

Luspatercept Meets Primary Endpoints in Phase 2 Alpha-Thalassemia Trial

Tim Smith joined the MJH Life Sciences team in 2022 and is currently an associate editor for HCPLive. He is also the executive producer of two podcasts: Skin of Color Savvy and The Medical Sisterhood. Previously, he was the producer of the Lungcast podcast. He graduated from UC Berkeley and worked in multimedia journalism as a staff writer prior to joining MJH. You can contact him at tsmith@mjhlifesciences.com.

 has granted premarket approval (PMA) to the Allurion Gastric Balloon System, which incorporates the Allurion Smart Capsule, for weight loss among those with obesity.

This February 23 announcement by Allurion Technologies, Inc. highlights the Smart Capsule’s patented technology, noting its intention to be swallowed within a brief, approximately 15-minute office visit. Internationally, the Smart Capsule has been implemented in more than 200,000 patients, the company noted, with data on its use documented in over 30 peer-reviewed medical journals.

“Obesity is a critical issue in the United States, and many patients are looking for alternatives or complements to existing options, including GLP-1 medications,” Shelby A. Sullivan, MD, professor of medicine at the Geisel School of Medicine at Dartmouth University and principal Investigator on the AUDACITY FDA pivotal trial, said in a statement.

Obesity is known to impact more than 100 million individuals in the US, with roughly 80 million falling within the body mass index (BMI) range of 30 - 40 kg/m².

 In this release, Allurion noted estimates suggesting around 20 million people in the US have initiated and subsequently discontinued GLP-1–based treatments. They also cited projections suggesting this figure may increase to 40 million by 2027.

In previous data, research cited by the Company suggests 65% of patients stop GLP-1 treatment within their initial year of use. This is most often attributed to adverse effects. Many can also experience rapid weight regain following the discontinuation of their use, they added.

To promote weight reduction in such individuals, the Allurion Gastric Balloon System is FDA-approved for short-term reduction among adults aged 22 - 65 years with obesity in the aforementioned BMI range. It is specifically for individuals who previously attempted and did not succeed with at least 1 structured weight loss program.

The Smart Capsule system does not require endoscopy, surgery, or anesthesia. Once the capsule is in a patient’s stomach, it is filled with liquid. It remains in place for about 4 months. Taking up space in the stomach, it promotes earlier satiety and diminishes one’s food intake. Following about 4 months, a proprietary Release Valve opens, leading the deflated device to pass naturally through one’s gastrointestinal tract.

Later, a second capsule may be administered 2 months following the first’s exit from the body. In contrast to anti-obesity medications, which Allurion noted must be received on a daily or weekly basis, a single Smart Capsule can allow for around 4 months of therapeutic effect from a single administration.

The submission of the Allurion Gastric Balloon System to the FDA for consideration followed the product’s successful topline data in the AUDACITY trial.

 The study, an open-label, multicenter, randomized controlled study, involved 550 participants who were randomized 1:1 to be given either 2 cycles of the Allurion Balloon plus care or moderate-intensity lifestyle therapy by itself.

The analysis met its responder co-primary endpoint, with 58% of Allurion Balloon recipients losing more than 5% of their total body weight at the 48-week mark (

 By this point in time, the mean difference in total body weight loss between the cohorts was shown by investigators to be 3.77%, corresponding to a 2.69% superiority margin.

This did not meet its pre-specified 3% margin required for the comparative co-primary endpoint (

 This was attributed in part to greater-than-anticipated weight loss in the control arm of the study. However, by Week 40, around the time the second balloon passed, the AUDACITY investigators noted the mean difference in weight loss as 4.22%, exceeding the 3% superiority threshold.

The reported rate of serious adverse events among those who were Allurion Balloon recipients was noted to be 3.1%.

 This was described as the lowest rate reported in an FDA pivotal trial of a liquid-filled intragastric balloon.

“The AUDACITY trial results coupled with the real-world experience outside the United States demonstrate the product’s safety and efficacy,” Sullivan said in her statement.

 “The solution offers an option that many people are looking for. It has a finite time in the body, is repeatable when necessary, and it does not require patients to take long-term medications for weight loss.”

Allurion Receives U.S. FDA Approval. Allurion Technologies, Inc. February 23, 2026. 

https://www.businesswire.com/news/home/20260223930098/en/Allurion-Receives-U.S.-FDA-Approval

Allurion Announces Positive Topline Results From AUDACITY Trial. Allurion Technologies, Inc. January 8, 2026. Accessed February 23, 2026. 

https://investors.allurion.com/news/news-details/2025/Allurion-Announces-Positive-Topline-Results-From-AUDACITY-Trial/default.aspx

FDA granted PMA to the Allurion Gastric Balloon System incorporating a swallowable Smart Capsule for short-term weight reduction in adults aged 22–65 years with BMI 30–40 kg/m² who failed at least one structured weight-loss program. The device is placed without endoscopy, surgery, or anesthesia, remains ~4 months, then deflates and passes naturally; a second cycle can follow after two months. Approval follows the 550-participant AUDACITY RCT showing significant responder benefit and 3.1% serious adverse events.

This approval of the Allurion Gastric Balloon System, incorporating the Allurion Smart Capsule, is indicated for weight loss management for those with obesity.

FDA News

Obesity Management

Obesity Management condition center page, resources on the topics of medical news and expert insight into clinical weight management can be found. Content includes articles, interviews, videos, podcasts, and breaking news on obesity research, treatment, and drug development.

FDA Grants Premarket Approval to Allurion Gastric Balloon System for Weight Loss

Ryan Livingston joined the MJH Life Sciences team in March of 2025, shortly after graduating from TCNJ with a bachelor’s degree in English. He enjoys creative writing, reading, and scuba diving. He can be reached at rlivingston@mjhlifesciences.com.

Martin Holst Lange, MD | Image Credit: Pharmacosmos

On February 23, 2026, Novo Nordisk announced that, despite demonstrating substantial weight loss after 84 weeks of treatment, CagriSema 2.4/2.4 mg failed to meet its primary endpoint of noninferiority compared to tirzepatide 15 mg.

“We are pleased with the weight loss of 23% for CagriSema in this open-label trial,” Martin Holst Lange, MD, executive vice president, R&D and chief scientific officer at Novo Nordisk, said in a statement. “CagriSema has the potential to be the first GLP-1/amylin-combination product to reach the market for people living with obesity, documenting that cagrilintide adds to the existing benefits of semaglutide and offers clinically meaningful additive weight loss effects superior to what has been observed with GLP-1 biology alone.”

CagriSema is a fixed-dose combination of cagrilintide 2.4, a long-acting amylin analogue, and semaglutide 2.4 mg. The drug induces 

 by reducing hunger and increasing feelings of fullness, helping people eat less and reduce their calorie intake. CagriSema is currently under investigation for overweight and obesity in the REDEFINE program and as a treatment for adults with 

REDEFINE is a phase 3 clinical development program comprised of 2 pivotal phase 3 trials including roughly 4600 adults with overweight or obesity. The program includes the following:

REDEFINE 1, an efficacy trial comparing CagriSema to placebo in patients with overweight or obesity without T2D, lasting 68 weeks and enrolling 3417 patients

REDEFINE 2, an efficacy trial comparing CagriSema to placebo in patients with T2D and either obesity or overweight, lasting 68 weeks and enrolling 1206 patients

REDEFINE 3, an event-driven cardiovascular outcomes trial of CagriSema versus placebo in patients with established cardiovascular disease with or without T2D, enrolling 7000 patients

REDEFINE 8, an efficacy trial comparing CagriSema to placebo in patients with obesity, lasting 104-weeks and enrolling 400 patients. This trial includes a 52-week extension phase investigating weight loss maintenance

REDEFINE 9, an efficacy trial comparing CagriSema to placebo in adults with obesity or overweight, lasting 68 weeks and enrolling 300 patients

REDEFINE 11, an efficacy trial of CagriSema compared to placebo in patients with obesity, lasting 80 weeks and enrolling 600 patients. This trial includes an 80-week extension phase investigating weight loss maintenance.

REDEFINE 4 was an 84-week open-label trial investigating CagriSema compared to tirzepatide, both of which were administered once weekly and subcutaneously. A total of 809 patients with obesity and ≥1 comorbidity, with a mean baseline body weight of 114.2 kg (251.77 lb), were enrolled in the trial.

Investigators found that, if all patients adhered to treatment, those receiving CagriSema 2.4 mg/2.4 mg saw a 23% weight loss after 84 weeks compared to 25.5% with tirzepatide. After applying the treatment regimen estimand, patients receiving CagrimSema saw weight loss of 20.2%, while tirzepatide recipients saw 23.6% weight loss.

Despite missing the primary endpoint of noninferiority, CagriSema did display a safe and well-tolerated profile. The most common adverse events were gastrointestinal, with the vast majority labelled as mild to moderate and diminishing over time.

“Based on the learnings from completed studies we look forward to the REDEFINE 11 readout, and the initiation of the higher-dose CagriSema trial, which are both designed to assess the full weight-loss potential of CagriSema,” Lange said. “The results in the REDEFINE program reinforce our commitment to transforming obesity care, through novel products such as CagriSema and zenagamtide with the potential to offer even greater health benefits for patients living with obesity.”

CagriSema has been submitted to the US Food and Drug Administration for weight management in December 2025, based on the REDEFINE 1 and REDEFINE 2 pivotal trials. According to the press release, Novo Nordisk anticipates a decision by late 2026.

Novo Nordisk. Novo Nordisk A/S: CagriSema demonstrated 23% weight loss in an open-label head-to-head REDEFINE 4 trial in people with obesity, the primary endpoint was not achieved. February 23, 2026. Accessed February 23, 2026. 

https://www.novonordisk.com/content/nncorp/global/en/news-and-media/news-and-ir-materials/news-details.html?id=916501

Hales CM. Expanding the Treat-to-Target Toolbox for Obesity and Diabetes Care. N Engl J Med. 2025;393(7):712-714. 

Novo Nordisk reported 84-week REDEFINE 4 results showing 23% weight loss with once-weekly CagriSema (cagrilintide 2.4 mg/semaglutide 2.4 mg) but failure to achieve noninferiority versus tirzepatide 15 mg. In the all-adherent estimand, weight loss was 23% vs 25.5%; under the treatment-regimen estimand, 20.2% vs 23.6%. Safety was consistent with incretin-based therapy, dominated by mild-to-moderate gastrointestinal events that waned over time. FDA submission (Dec 2025) anticipates a late-2026 decision.

The phase 3 REDEFINE 4 trial saw CagriSema achieve 23% weight loss, but fail to demonstrate noninferiority to tirzepatide in patients with overweight or obesity.

Endocrinology

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CagriSema Demonstrates Weight Loss, Fails to Achieve Primary Endpoint Compared to Tirzepatide

Abigail Brooks is an assistant managing editor for 

She joined MJH Life Sciences in August 2023 shortly after graduating from Monmouth University where she earned her BA in Communication with a concentration in Public Relations/Journalism and later an MA in Interactive Digital Media. She enjoys traveling, running, and reading books. She can be reached at abrooks@mjhlifesciences.com.

The US Food and Drug Administration has granted Fast Track and Orphan Drug designations to TSRA-196, an 

 gene editing program from Tessera Therapeutics, for the treatment of adults with 

 who are homozygous for the PiZ allele (PiZZ).

As described in a February 23, 2026, press release from the Company, TSRA-196 is being jointly developed with Regeneron and is designed to precisely correct the genetic mutation underlying AATD, with the goal of restoring production of functional alpha-1 antitrypsin (AAT) protein through a one-time, durable treatment option for patients.

“Despite a clear understanding of the mutation that drives severe AATD, options are limited to life-long treatments that fail to address the underlying cause of the disease,” said David Davidson, MD, Chief Development and Medical Officer of Tessera Therapeutics.

“Based on compelling evidence to date, we believe that TSRA-196 has the potential to permanently correct the underlying mutation responsible for this debilitating disease and address a high unmet need for a durable therapy. Fast Track and Orphan designations allow us to accelerate the development of TSRA-196 to dramatically improve the lives of people living with AATD, and we look forward to translating this promising mechanism into meaningful outcomes in our ongoing global first-in-human trial.”

In January, the FDA cleared Tessera’s Investigational New Drug (IND) application for TSRA-196 in AATD. The Company also received Australian Human Research Ethics Committee (HREC) approval to begin the phase 1/2 clinical trial to assess the safety, tolerability, and efficacy of TSRA-196 in adults with AATD.

The first-in-human, open-label, multi-national study will administer a single intravenous administration of TSRA-196 and follow participants longitudinally for safety and key biomarkers relevant to AAT expression and function.

Previous preclinical data presented at the American Society of Gene & Cell Therapy 28th Annual Meeting highlighted durable, high-fidelity genome editing of SERPINA1, the locus responsible for AATD, in mice and non-human primates following a single dose of TSRA-196, with high liver editing specificity, no germline or off-target editing, and favorable safety and tolerability using Tessera’s proprietary lipid nanoparticle delivery vehicle. These findings reinforce TSRA-196’s potential to correct the underlying genetic cause of AATD and support its advancement into clinical development.

Despite not yet having any FDA-approved treatments, the therapeutic landscape for AATD is greatly expanding. In addition to TSRA-196’s Fast Track and Orphan Drug designations, YOLT-202, an investigational 

 base editing therapy from YolTech Therapeutics, recently showed positive safety and tolerability as well as meaningful increases in AAT levels in evaluated patients treated with the 35 mg and 45 mg dose levels in an investigator-initiated trial.

Tessera Therapeutics. Tessera Therapeutics Receives U.S. FDA Fast Track and Orphan Drug Designations for its Lead In Vivo Gene Editing Program TSRA-196 for the Treatment of Adults with AATD. February 23, 2026. Accessed February 23, 2026. 

https://www.tesseratherapeutics.com/news/tessera-therapeutics-receives-u-s-fda-fast-track-and-orphan-drug-designations-for-its-lead-in-vivo-gene-editing-program-tsra-196-for-the-treatment-of-adults-with-aatd

Tessera Therapeutics. Tessera Therapeutics Announces FDA Clearance of IND Application for its Lead In Vivo Gene Editing Program TSRA-196 for AATD. January 12, 2026. Accessed February 23, 2026. 

https://www.tesseratherapeutics.com/news/tessera-therapeutics-announces-fda-clearance-of-ind-application-for-its-lead-in-vivo-gene-editing-program-tsra-196-for-aatd

Tessera Therapeutics. Regeneron and Tessera Therapeutics to Jointly Develop TSRA-196, an Investigational Gene Editing Therapy for Alpha-1 Antitrypsin Deficiency (AATD). December 1, 2025. Accessed February 23, 2026. 

https://www.tesseratherapeutics.com/news/regeneron-and-tessera-therapeutics-to-jointly-develop-tsra-196-an-investigational-gene-editing-therapy-for-alpha-1-antitrypsin-deficiency-aatd

Brooks A. Single Dose YOLT-202 Gene-Editing Therapy Increases Functional AAT Levels in AATD. HCPLive. February 19, 2026. Accessed February 23, 2026. 

https://www.hcplive.com/view/single-dose-yolt-202-gene-editing-therapy-increases-functional-aat-levels-in-aatd

The FDA granted Fast Track and Orphan Drug designations to TSRA-196, an in vivo gene-editing candidate for adults with severe alpha-1 antitrypsin deficiency who are PiZZ. Co-developed by Tessera Therapeutics and Regeneron, TSRA-196 aims to precisely correct the SERPINA1 mutation to restore functional AAT production via a one-time, durable intervention. An IND has been cleared and a global, open-label phase 1/2 study is underway using single IV dosing with longitudinal safety and biomarker follow-up.  

The FDA has granted both designations to TSRA-196, an in vivo gene editing program from Tessera Therapeutics, for the treatment of PiZZ alpha-1 antitrypsin deficiency.

Hepatology

Alpha-1 antitrypsin deficiency

TSRA-196 Gets FDA Fast Track, Orphan Drug Designations for AATD

Chelsie Derman is an associate editor for 

who covers psychiatry, sleep medicine, geriatrics, migraine, and allergy. She joined MJH Life Sciences in September 2023 after graduating from The College of New Jersey with a bachelor’s degree in journalism and professional writing. In her free time, she enjoys creative writing and reading.

FDA clears iloperidone metabolite, milsaperidone, for acute bipolar I disorder and schizophrenia, expanding treatment options.

Psychiatry

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FDA Approves Milsaperidone for Acute Bipolar I Disorder, Schizophrenia

Diana Isaacs, PharmD, is an endocrine clinical pharmacist, director of Education and Training in Diabetes Technology, and codirector of Endocrine Disorders in Pregnancy at the Cleveland Clinic.

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Cohosts Diana Isaacs, PharmD, and Natalie Bellini, DNP, discuss the recent release of Eli Lilly's Zepbound as an injectable with the KwikPen.

podcasts page features a feed of medical news and interview podcast series, including DocTalk, Lungcast, Derm Discussions, and Heart Trials.

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Diabetes Dialogue: Technology, Therapeutics, & Real-World Perspectives is a monthly podcast hosted by Diana Isaacs, PharmD, and Natalie Bellini, DNP, aimed at providing a regular roundup of the latest clinically applicable insights across diabetes and metabolic diseases, with a focus on leveraging technology to improve care.

Strategic Alliance Partnership

Diabetes Dialogue: Launch of the Zepbound KwikPen

Frances Lee, MD, is an assistant professor of medicine in the division of liver diseases at Mount Sinai.

Videos

New studies reveal potential benefits of alfagimor in reducing fatigue and pruritus, highlighting its role in second-line therapy for patients.

New Fatigue Signals From Elafibranor: Results From the ELATIVE Phase III Trial

In this episode, experts delve into the limited treatment options to address the symptom of fatigue in patients with primary biliary cholangitis. They highlight how small changes, like regular sleep schedules and exercise, can improve fatigue.

Addressing Fatigue: Traditional Management Outside Primary Therapy

The CUPID-C randomized clinical trial confirmed CUPID-A results, with dupilumab significantly reducing urticaria in anti-IgE–naive individuals with CSU.

Urticaria

The HCPLive urticaria page is a comprehensive resource for clinical news and insights on chronic idiopathic urticaria and chronic spontaneous urticaria. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for urticaria, and more.

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Dupilumab Reduces Urticaria Activity in Anti-IgE–Naive Patients with CSU

Levine discusses 24-hour adverse event resolution, retreatment questions, and scalability of supervised psilocybin therapy for treatment-resistant depression.

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COMP360 Safety Data in Phase 3 TRD Trials, With Steve Levine, MD

Ron Vender, MD, is a dermatologist in Hamilton, Ontario, and is the founder and director of Dermatrials Research Incorporated and Venderm Consulting Inc. He is also an associate clinical professor at McMaster University in the Department of Medicine, Division of Dermatology.

This interview segment highlights additional points on bimekizumab and psoriasis related to patient-reported outcomes (PROs) in the 3 year BE RADIANT study.

Plaque Psoriasis

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Skin Clearance and Psoriasis Improvements from Bimekizumab, With Ron Vender, MD

Diana Isaacs, PharmD, and Natalie Bellini, DNP, discuss the Portal implanted insulin pump, which has received Breakthrough Device designation from the FDA.

Type 2 Diabetes

Diabetes Dialogue: Implantable Insulin Pumps and Interoperability

The HCPFive: Top News for Healthcare Providers from the Week of 02/15

The new joint guidelines include a clinical classification system, major contributing factors, and recommendations for treatment and follow-up.

Cardiology

Cardiology condition center page is a comprehensive resource for clinical news and insights on cardiovascular and cardiometabolic diseases. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for heart disease and cardiovascular events, as well as associated diabetes, renal failure, and more...

Venous Thromboembolism

The HCPLive venous thromboembolism page is a resource for medical news and expert insights on VTE. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for thrombotic disease, and more.

Latest News

PROSERA: Seralutinib Falls Short in Pulmonary Arterial Hypertension Phase 3 Trial

Luspatercept Meets Primary Endpoints in Phase 2 Alpha-Thalassemia Trial

FDA Grants Premarket Approval to Allurion Gastric Balloon System for Weight Loss

CagriSema Demonstrates Weight Loss, Fails to Achieve Primary Endpoint Compared to Tirzepatide

TSRA-196 Gets FDA Fast Track, Orphan Drug Designations for AATD