FKBP1A

BALTIMORE, Oct. 17, 2024 (GLOBE NEWSWIRE) -- Rapafusyn Pharmaceuticals, a leader in the field of non-degrading molecular glues, is pleased to announce the upcoming presentation of preclinical in vivo data from its selective ENT1 inhibitor for Acute Kidney Injury (AKI) at the American Society for Nephrology (ASN) Kidney Week 2024 to be held from October 24-27, 2024, in San Diego, CA. Rapafusyn’s lead program, RAP-0001, is advancing rapidly to address a critical unmet need: the prevention of Acute Kidney Injury (AKI) in patients undergoing cardiac surgery. AKI is a severe and common complication, leading to prolonged hospital stays, increased morbidity, and higher mortality rates. With no approved therapies currently available, novel solutions are urgently needed. RAP-0001, a potent and selective inhibitor of the ENT1 transporter, has shown preclinical efficacy in preventing ischemia/reperfusion injury, a major driver of AKI. “This compound was discovered through the RapaGlue™ platform. As a molecular glue, RAP-0001 uniquely binds to the intracellular chaperone protein FKBP12 and ENT1 to form a ternary complex. To our knowledge, it’s the only SLC inhibitor that targets the intracellular domain of a transporter,” said Dr. Matthew Olson, VP of Biological Sciences of Rapafusyn, who will present in vivo data demonstrating RAP-0001’s ability to significantly reduce kidney damage in this high-risk context on October 25 th , 2024. Dr. Sean Hu, CEO of Rapafusyn, stated, “We believe RAP-0001 could change the standard of care for AKI, providing a critical therapeutic option where none currently exist. This is yet another example of recent developments demonstrating how non-degrading molecular glues could potentially tackle challenging disease targets and address high unmet medical needs in ways more effective than other drug modalities.” About Rapafusyn At Rapafusyn, we’re creating non-degrading molecular glues to address hard-to-drug targets. Using our FKBP12-binding macrocyclic peptide platform, we’ve built large DNA encoded libraries (DELs) and arrayed libraries, yielding novel starting points. Our approach targets intracellular proteins and domains of transmembrane proteins like SLCs, ion channels, and GPCRs. With a modular design, we rapidly optimize key drug properties to accelerate drug-discovery. For more information, please visit or contact Heather Lavin at hlavin@rapafusyn.com .

HOUSTON, June 28, 2021 (GLOBE NEWSWIRE) -- Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers, today announced it has entered into a license agreement with the University of North Carolina Lineberger Comprehensive Cancer Center (UNC Lineberger) and Massachusetts General Hospital (Mass General) covering certain intellectual property and technology rights regarding the company’s CaspaCIDe (inducible caspase-9, or iC9) safety switch and related technologies, and the use of rimiducid. CaspaCIDe may facilitate the use of cell therapies where cytokine release syndrome and neurotoxicities have been observed, in the pursuit of novel targets with on-target/off-tumor safety concerns, and in conjunction with next generation higher potency cell therapy constructs. This agreement with UNC Lineberger and Mass General covers four CAR-T programs incorporating Bellicum’s technology currently in development, two of which are owned by UNC Lineberger and two of which are co-owned by both institutions. Under this agreement, upon out-license of each program to an industry partner, Bellicum will receive an upfront payment and be entitled to a percentage of certain consideration paid to the institutions by the industry partner and a single digit percent royalty on the global sales of the product. Additional details of the financial arrangements are not disclosed. “We are thrilled by the opportunity to expand the impact of our CaspaCIDe technology to benefit patients through this agreement with leading oncology research and treatment centers of excellence,” said Rick Fair, President and CEO of Bellicum Pharmaceuticals. “We believe the agreement reflects the potential value of our switch technology, which may enhance the benefit/risk profile of cell therapies. We continue to incorporate the technology into our internal programs and intend to make it more broadly available via external collaborations.” “The unique inducible caspase-9 technology covered by this agreement has the potential to improve the safety profile of cellular immunotherapies, reduce the risk of serious adverse events, and improve patient outcomes,” stated Gianpietro Dotti, M.D., Co-leader, Immunology Program, UNC Lineberger. “We are pleased to incorporate Bellicum’s CaspaCIDe into four of our promising cell therapy constructs.” About CaspaCIDe® CaspaCIDe (inducible caspase-9, or iC9) is Bellicum’s chemical induction of dimerization (CID) safety switch technology designated to eliminate cells in the event of toxicity. The CaspaCIDe switch consists of the CID-binding domain coupled to the signaling domain of caspase-9, an enzyme that is part of the apoptotic pathway. Infusion of rimiducid is designed to trigger activation of this domain of caspase-9, which in turn leads to selective apoptosis of the CaspaCIDe-containing cells. In clinical studies, use of CaspaCIDe has resulted in clinical improvement in most patients as early as 24 hours after rimiducid administration. Further, because CaspaCIDe is designed to be permanently incorporated into Bellicum’s cellular therapies, the safety switch has the potential to be available for use when needed long after the initial therapy is delivered. About Bellicum Pharmaceuticals Bellicum is a clinical stage biopharmaceutical company striving to deliver cures through controllable cell therapies. The company’s next-generation product candidates are differentiated by powerful cell signaling technologies designed to produce more effective CAR-T cell therapies. Bellicum’s GoCAR-T® product candidates, BPX-601 and BPX-603, are designed to be more efficacious CAR-T cell products capable of overriding key immune inhibitory mechanisms. More information about Bellicum can be found at . Forward-Looking Statements This press release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Bellicum may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “designed,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Forward-looking statements include statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: the potential for the Bellicum’s safety switch technology to improve the benefit/risk profile of cell therapies; the potential for expanded beneficial impact of the CaspaCIDe technology on patients enabled by the license agreement; Bellicum’s plans to make our technology more broadly available via external collaborations; and the potential useful life of the CaspaCIDe safety switch. Various factors may cause differences between Bellicum’s expectations and actual results as discussed in greater detail under the heading “Risk Factors” in Bellicum’s filings with the Securities and Exchange Commission, including without limitation Bellicum’s quarterly report on Form 10-Q for the three months ended March 31, 2021 and Bellicum’s annual report on Form 10-K the year ended December 31, 2020. Any forward-looking statements that Bellicum makes in this press release speak only as of the date of this press release. Bellicum assumes no obligation to update our forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release. Source: Bellicum Pharmaceuticals Investors:Robert H. UhlManaging DirectorWestwicke ICR858-356-5932Robert.uhl@westwicke.com