EU Approves Novel Antisense Oligonucleotide Therapy for Hereditary Angioedema; Think Bioscience Secures $55M to Target 'Undruggable' Proteins

Innovative Antisense Oligonucleotide Therapy Receives EU Approval

Ionis Pharmaceuticals and Otsuka Pharmaceutical Co., Ltd. announced today that the European Commission (EC) has approved Dawnzera (donidalorsen) for marketing in the European Union, intended for routine prevention of recurrent hereditary angioedema (HAE) attacks in adults and adolescents aged 12 years and older. This approval is based on the positive opinion previously issued by the Committee for Medicinal Products for Human Use (CHMP).

This approval is based on the positive results of the Phase 3 OASIS-HAE and OASISplus studies. Dawnzera demonstrated positive efficacy across multiple disease assessment metrics, including significantly and consistently reducing the average monthly attack rate of HAE.In the OASISplus open-label extension study, the overall average monthly seizure rate decreased by 94% after one year of treatment.Dawnzera is a ligand-conjugated antisense oligonucleotide (LICA) drug designed to silence the expression of prekallikrein (PKK) and interrupt the relevant signaling pathways that lead to HAE attacks.

New Player Completes $55 Million Series A Financing for "Undruggable" Targets

Think Bioscience, a biotechnology company focused on cracking challenging drug targets, has recently completed a $55 million oversubscribed Series A financing round.Think Bioscience uncovers innovative protein-binding "pockets" for drug chemistry through synthetic biology, enabling the development of small-molecule drugs for historically challenging targets.Its leading program targets mutants that cause Noonan syndrome, a genetic disorder often accompanied by life-threatening cardiac and lymphatic system issues, short stature, cognitive impairment, and various chronic symptoms such as pain. Currently, there are no approved therapies that can address the underlying biological mechanisms of the disease.

Despite recent advances in computational chemistry, most proteins in the human proteome are still considered "undruggable," meaning they lack obvious "pockets" for drug binding.Think BioscienceThe synthetic biology platform discovers previously overlooked "pockets" through high-throughput functional screening, advancing the development of small-molecule drugs.The platform has expanded to various target types, including transcription factors, protein phosphatases, kinases, proteases, and GTPases.

Off-the-Shelf CAR-T Therapy Receives FDA Breakthrough Therapy Designation

Wugen, Inc. today announced that its investigational off-the-shelf CAR-T cell therapy, sofi-cel, has been granted Breakthrough Therapy Designation by the U.S. FDA for the treatment of hematologic malignancies of T-cell origin.Sofi-cel is a potential "first-in-class" allogeneic CAR-T cell therapy targeting CD7.It aims to overcome the technical challenges faced in using CAR-T cells for treating T-cell tumors. Wugen employs gene editing technology to knock outCD7Genes and T-cell receptor alpha constant region (TRAC) gene, thereby preventing CAR-T cells from fratricide and reducing the risk of graft-versus-host disease (GvHD).

Sofi-cel is currently being evaluated in a pivotal clinical trial (T-RRex) for the treatment of patients with relapsed or refractory (R/R) T-cell acute lymphoblastic leukemia or T-cell lymphoblastic lymphoma (T-ALL/LBL).

References:

[1] Think Bioscience Raises $55M in Oversubscribed Series A. Retrieved January 21, 2026, from https://www.prnewswire.com/news-releases/think-bioscience-raises-55m-in-oversubscribed-series-a-302665119.html?tc=eml_cleartime

[2] DAWNZERA™ (donidalorsen) approved in the European Union for hereditary angioedema (HAE). Retrieved January 21, 2026, from https://ir.ionis.com/news-releases/news-release-details/dawnzeratm-donidalorsen-approved-european-union-hereditary 

[3] U.S. FDA Grants to Wugen’s WU-CART-007 Breakthrough Therapy Designation for Treatment of Relapsed or Refractory T Cell Acute Lymphoblastic Leukemia / T Cell Lymphoblastic Lymphoma. Retrieved January 21, 2026, from http://cts.businesswire.com/ct/CT?id=ftfLlWd9aZbF2NXfwppam3Bd664=&newsLang=en&newsId=20260121930614÷=1990896910

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