Be Bio Raised $82 Million do Advance Engineered Cell Therapy for Hemophilia B

Rare Daily Staff

Be Biopharma said it raised $82 million to advance into the clinic its lead program BE-101, an engineered cell therapy to treat hemophilia B.

The company also unveiled its second development candidate for hypophosphatasia, which like its lead candidate also utilizes gene editing to engineer B cells to produce sustained levels of therapeutic proteins.

The financing included investment from ARCH Venture Partners, Atlas Venture, RA Capital Management, Alta Partners, Longwood Fund, Bristol Myers Squibb, and Takeda Ventures.

Hemophilia B is an X-linked recessive bleeding disorder. It is caused by mutations in the gene that encodes for the FIX protein, an essential enzyme in the coagulation cascade. This can lead to spontaneous bleeding as well as bleeding following injuries or surgery. People with hemophilia B bleed longer than other people. Bleeds can occur internally, into joints and muscles, or externally, from minor cuts, dental procedures or trauma.

The current standard of care remains prophylactic administration FIX replacement therapy with a dosing frequency that ranges from every week to every 2 weeks. The short biological half-life of FIX requires lifelong frequent infusions to maintain therapeutic levels.

BE-101 is a first-in-class B cell medicine that is engineered to insert the human factor IX (FIX) gene into primary human B cells, allowing for continuous expression of active FIX for the treatment of hemophilia B. BE-101 has the potential to express sustained therapeutic FIX activity levels with a single infusion while having the flexibility to be titrated and/or re-dosed, and without the need for preconditioning. The U.S. Food and Drug Administration granted BE-101 Fast Track designation.

The company has begun enrolling its phase 1/2 trialfor BE-101.

The potential to maintain therapeutic FIX activity levels while reducing dosing frequency associated with current FIX replacement regimens could address the considerable infusion burden associated with current therapies and potentially drive reductions in the annualized bleeding rates and FIX usage, the company said.

“Advancing two BCMs that harness the potential of this new modality represents an exciting step in transforming the treatment landscape for their respective indications with potentially best-in-class genetic medicines,” said Joanne Smith-Farrell, CEO of Be Bio